Lacosamide for neonatal seizures

Kaur M, Utidjian L, Abend NS, Dickinson K, Roebling R, McDonald J, Maltenfort MG, Foskett N, Elmoufti S, Guerriero RM, Jain BG, Pajor NM, Rao S, Shellhaas RA, Slaughter L, Forrest CB. Retrospective Multicenter Cohort Study on Safety and Electroencephalographic Response to Lacosamide for Neonatal Seizures. Pediatr Neurol. 2024 Jun;155:18-25. doi: 10.1016/j.pediatrneurol.2024.03.007. Epub 2024 Mar 13. PMID: 38579433.

Abstract

Background: There is growing evidence supporting the safety and effectiveness of lacosamide in older children. However, minimal data are available for neonates. We aimed to determine the incidence of adverse events associated with lacosamide use and explore the electroencephalographic seizure response to lacosamide in neonates.

Methods: A retrospective cohort study was conducted using data from seven pediatric hospitals from January 2009 to February 2020. For safety outcomes, neonates were followed for ≤30 days from index date. Electroencephalographic response of lacosamide was evaluated based on electroencephalographic reports for ≤3 days.

Results: Among 47 neonates, 98% received the first lacosamide dose in the intensive care units. During the median follow-up of 12 days, 19% of neonates died, and the crude incidence rate per 1000 patient-days (95% confidence interval) of the adverse events by diagnostic categories ranged from 2.8 (0.3, 10.2) for blood or lymphatic system disorders and nervous system disorders to 10.5 (4.2, 21.6) for cardiac disorders. Electroencephalographic seizures were observed in 31 of 34 patients with available electroencephalographic data on the index date. There was seizure improvement in 29% of neonates on day 1 and also in 29% of neonates on day 2. On day 3, there was no change in 50% of neonates and unknown change in 50% of neonates.

Conclusions: The results are reassuring regarding the safety of lacosamide in neonates. Although some neonates had fewer seizures after lacosamide administration, the lack of a comparator arm and reliance on qualitative statements in electroencephalographic reports limit the preliminary efficacy results.

Chourasia N, Dohmeier J, Curry J, Parkhurst S, Mudigoudar B, Rivas-Coppola M, Wheless J. Clinical Experience With Lacosamide as an Adjunct Treatment for Neonatal Seizures: A Retrospective Single-Center Study. Pediatr Neurol. 2024 Aug;157:134-140. doi: 10.1016/j.pediatrneurol.2024.05.019. Epub 2024 Jun 5. PMID: 38917517.

Abstract

Background: Lacosamide (LCM) is a third-generation antiseizure medication (ASM) currently approved for the treatment of focal seizures in children aged greater than one month. There are limited data on its efficacy in the neonatal age group. We describe our experience with LCM as an adjunct ASM for the treatment of neonatal seizures.

Methods: A retrospective chart review over a five-year period (2018 to 2022) was conducted at Le Bonheur Children's Hospital to identify neonates with electroencephalography (EEG)-proven seizures who were treated with LCM. Data were collected on electroclinical seizure characteristics, underlying etiology, ASMs, treatment response, and any adverse effects.

Results: A total of 15 neonates with EEG-confirmed seizures who were treated with LCM were included. Ten neonates achieved seizure cessation after LCM was added to their ASM regimen consisting of phenobarbital, levetiracetam, or both. No new treatment-related adverse effects were noted.

Conclusions: LCM is effective as an adjunct treatment for neonatal seizures. Randomized controlled studies are needed to establish its effectiveness and adequate dosing regimen in this population.

Jewell TI, Carrasco M, Hsu DA, Knox AT. Lacosamide Boluses Decreased Seizure Burden and Were Well Tolerated in Neonates With Acute Seizures: A Single-Center Retrospective Case Series. J Child Neurol. 2025 Feb;40(2):116-122. doi: 10.1177/08830738241286108. Epub 2024 Oct 14. PMID: 39397495; PMCID: PMC11781981.

Abstract

Introduction: Neonatal seizures are associated with worsened neurodevelopmental outcomes. Phenobarbital, the only US Food and Drug Administration (FDA)-approved treatment for neonatal seizures, can cause neuronal apoptosis and may worsen neurodevelopmental outcomes. Lacosamide may be an efficacious treatment for neonatal seizures. Methods: We assessed the impact of lacosamide boluses on seizure burden in a retrospective cohort of 15 neonates monitored with video electroencephalography (EEG). Medication bolus times and seizure start/end times on EEG tracings determined change in seizure burden. Results: Seven patients received lacosamide as first- or second-line treatment and 8 as third-line or later. Average 4-hour seizure burden decreased from 13% to 3% following lacosamide boluses (P = .002). Reduction in seizure burden greater than 30% followed 79% of boluses. Lacosamide was well tolerated; one patient experienced mild asymptomatic episodic bradycardia that medication taper resolved. Conclusions: Lacosamide significantly decreased seizure burden in this cohort. Prospective studies of lacosamide treatment for neonatal seizures are warranted.

Cortes-Ledesma C, Arias-Vivas E, Ruiz-Ocaña de Las Cuevas G, Santana-Cabrera E, Garcia-Ron A. Clinical Impact and Safety Profile of Intravenous Lacosamide Administration as Adjunctive Therapy for Neonatal Seizures. J Child Neurol. 2025 Apr;40(4):291-295. doi: 10.1177/08830738241296177. Epub 2024 Nov 14. PMID: 39539162.

Abstract

The aim of this retrospective descriptive study was to evaluate the clinical impact and safety profile of lacosamide in neonates with symptomatic refractory seizures.Patients diagnosed with symptomatic refractory seizures who received lacosamide were included in the study. Follow-up assessments were conducted until 24 months of age, during which data on lacosamide dosage, duration of exposure, concurrent treatments, and potential side effects were collected. A total of eight patients were enrolled, with lacosamide administered as a third- or fourth-line treatment for symptomatic refractory seizures. Following loading dose, 62.5% of the patients achieved complete remission of seizure activity without recurrence. In the remaining cases, a reduction in seizure frequency was observed. No adverse effects attributable to lacosamide were reported.In conclusion, lacosamide may be effective in achieving seizure remission in newborns with symptomatic refractory seizures, and all patients demonstrate excellent tolerance. Brief exposure to lacosamide was sufficient, and no adverse effects were observed up to 24 months of age. However, randomized controlled trials are necessary to confirm these findings.

Neuralink to treat amyotrophic lateral sclerosis

Brad Smith, an Arizona husband and father with ALS, has become the third person to receive Neuralink, the brain implant made by Elon Musk’s company.

He is also the first ALS patient and the first non-verbal person to receive the implant, he shared in a post on X on Sunday.

"I am typing this with my brain. It is my primary communication," Smith, who was diagnosed in 2020, wrote in the post, which was also shared by Musk. He went on to thank Musk.

Smith is completely paralyzed and relies on a ventilator to breathe. He created a video using the brain-computer interface (BCI) to control the mouse on his MacBook Pro, he stated. 

"This is the first video edited with [Neuralink], and maybe the first edited with a BCI," he said. 

"Neuralink has given me freedom, hope and faster communication."

The video was narrated by Smith’s "old voice," he said, which was cloned by artificial intelligence from recordings before he lost the use of his voice. 

"I want to explain how Neuralink has impacted my life and give you an overview of how it works," he said.

ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease, is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, according to The ALS Association. 

Over time, the disease impairs muscle control until the patient becomes paralyzed. ALS is ultimately fatal, with an average life expectancy of three years, although 10% of patients can survive for 10 years and 5% live 20 years or longer.

It does not impact cognitive function.

Neuralink, which is about 1.75 inches thick, was implanted in Smith’s motor cortex, the part of the brain that controls body movement.

The implanted device captures neuron firings in the brain and sends a raw signal to the computer.

"AI processes this data on a connected MacBook Pro to decode my intended movements in real time to move the cursor on my screen," Smith said.

"Neuralink has given me freedom, hope and faster communication," he added. "It has improved my life so much. I am so happy to be involved in something big that will help many people." 

Smith is also a man of faith, saying that he believes God has put him in this position to serve others. 

"I have not always understood why God afflicted me with ALS, but with time, I am learning to trust His plan for me," he said. 

"God loves me and my family. He has answered our prayers in unexpected ways. He has blessed my kids and our family. So I'm learning to trust that God knows what he is doing."

Smith also said he is grateful that he gets to work with the "brilliant people" at Neuralink and do "really interesting work."

"Don't get me wrong, ALS still really sucks, but I am talking about the big picture," he said. "The big picture is, I am happy." 

Dr. Mary Ann Picone, medical director of the MS Center at Holy Name Medical Center in Teaneck, New Jersey, applauded Neuralink's capabilities.

"This is an amazing development that now the third person to use Neuralink has gained the ability with the use of AI to type with neural thoughts," Picone, who was not involved in Smith’s care, told Fox News Digital. 

"The now-realized potential of Neuralink is to allow patients with quadriplegia to control computers and mobile devices with their thoughts." 

There are some risks involved with the implant, Picone noted. These include surgical infection, bleeding and damage to the underlying brain tissue.

"But the benefits are that patients who are paralyzed would have the potential to restore personal control over the limbs by using their thoughts," she said.  

Dr. Peter Konrad, M.D., Ph.D., chairman of the department of neurosurgery at WVU Rockefeller Neuroscience Institute in West Virginia, called Neuralink a "remarkable demonstration of the power of AI-driven technology."

"Mr. Smith is an incredible hero for those who are severely disabled from diseases such as ALS," Konrad, who also was not involved in Smith’s care, told Fox News Digital.

"Mr. Smith is an incredible hero for those who are severely disabled from diseases such as ALS."

Konrad also spoke of the advancements that have occurred since the past generations of BCI technology.

"It is encouraging to see faster progress being made with neural devices reaching clinical trials in the past five to 10 years," he said. "However, we are still awaiting development of a BCI device that does not require a team of engineers and experts to customize each and every severely disabled patient with this technology."

"For every Brad Smith out there, there are hundreds of thousands of other disabled patients awaiting access to this technology," he said.

"This video demonstrates the safety of these types of devices — now it’s time to provide larger access to these devices through a new generation of educated physicians, engineers and manufacturers able to deploy this technology."

https://www.foxnews.com/health/paralyzed-man-als-third-receive-neuralink-implant-can-type-brain

Mammillary body and hippocampal injury after acute perinatal arterial ischemic stroke

Lequin, Maarten, Baak, Lisanne M., Dudink, Jeroen, Vann, Seralynne, Groenendaal, Floris, de Vries, Linda S., Benders, Manon J.N.L., Nijboer, Cora H.A., Wagenaar, Nienke and van der Aa, Niek E. 2025. Mammillary body and hippocampal injury after acute perinatal arterial ischemic stroke. Neurology Open Access 1 (1) , e0004. 10.1212/wn9.0000000000000004

Abstract

Background and Objectives
Perinatal arterial ischemic stroke (PAIS) affects approximately 1–2 per 5,000 full-term births and can result in long-term neurologic changes such as hemiparesis, epilepsy, and cognitive impairments, including memory difficulties. The mammillary bodies (MBs) and the hippocampi (HCs) are both important brain structures for memory, and they are affected in infants with perinatal asphyxia, but less is known about their involvement in PAIS. We performed a retrospective study to determine the prevalence of MB and HC injury and to assess whether the injury varied according to the arterial territory involved and the co-occurrence of perinatal asphyxia.

Methods
This retrospective study assessed the status of the MB and HC of 130 neonates born between 2005 and 2023 diagnosed with acute PAIS based on neonatal MRI. Involvement of the MB and HC was based on diffusion-weighted imaging and T1/T2-weighted imaging. The hemisphere and arterial territory involved in the PAIS were documented along with clinical measures and the presence of perinatal asphyxia and therapeutic hypothermia.

Results
MRI scans were acquired at a median of 5 days after birth. Most of the strokes involved the middle cerebral artery. The MB could be assessed in 127 of 130 patients with PAIS and were found to be injured in 21 (16.5%) of 127, with equivocal signal change in a further 27 (21%) of 127 patients. MB injury occurred more often with larger strokes and was almost exclusively found bilaterally. MB injury was more prevalent when there was co-occurring perinatal asphyxia (odds ratio 3.56). Only 6% (8/130) of patients with PAIS showed HC injury, which was typically unilateral and located within the primary arterial stroke area.

Discussion
MB injury is frequently present in neonates with PAIS, while HC injury occurs less often. The MB and HC injuries seem to be caused by different processes, with the HC injury typically resulting from the direct impact of the stroke. Understanding the mechanisms through which the MBs become injured in PAIS, both with and without perinatal asphyxia, is an important future goal.

Canine diagnosis

Breanna Bortner’s dog, Mochi, began fixating on her breast before she was diagnosed with stage 2B triple-negative breast cancer, showing unusual and intuitive behavior throughout her journey.

In 2023, Bortner — who now runs the blog "Brave Beautiful Boobies" documenting her experience — discovered a lump in her breast.

There were about six weeks between the discovery of the lump and the biopsy, she recalled.

During this time, Bortner's cockapoo, Mochi, became fixated on the breast with the lump. The behavior came as a surprise to Bortner, who said the dog had never behaved that way before.

"Even when I had been sick in the past or dealt with other things, he had never done that, so that was the first time that I really noticed how in tune he was to my body," she said.

The results of the biopsy soon confirmed that Bortner had breast cancer — but she said she already knew, courtesy of Mochi.

"I was like, oh my gosh, he’s known this whole time. Before I knew and the doctors knew."

Doctors diagnosed Bortner with stage 2B triple-negative invasive ductal carcinoma, a type of breast cancer that has fewer treatment options than other types of invasive breast cancer, according to the American Cancer Society.

This is because these cancer cells do not have certain receptors that allow them to be treated with hormone therapy.

"If the cancer has not spread to distant sites, surgery is an option. Chemotherapy might be given first to shrink a large tumor, followed by surgery," reads the ACS website.

After doctors confirmed the cancer, Bortner said the process moved very quickly. She underwent five-and-a-half months of chemotherapy (16 rounds total).

"Chemotherapy is a very scary, daunting word," she said. "Everything about [chemotherapy drugs] is scary. I found that calling them ‘healing sessions’ instead of chemotherapy was better for me."

Bortner said that Mochi, who she calls her "soul dog," continued to be a source of comfort — and a reason to keep going in those dark days.

"At the time, he was only 2 years old," she said.

"And I was like, ‘I cannot leave this earth before you do.’ Like, no way. ‘I should be the one burying you. You shouldn't be the one having to lose your human mom.’"

Tasks as mundane as feeding the dog or taking him on a walk helped Bortner get off the couch and keep going.

Mochi learned to adapt to Bortner's new lifestyle during recovery, as their walks were slower and shorter. 

When Bortner would nap for long periods or sometimes cry, the dog was always there to comfort her, she shared.

When she left for appointments — sometimes multiple times per day — Mochi would dig through the trash to collect his owner's hair, which was falling out at the time due to chemotherapy.

"He would dig out my hair to smell me," Bortner said.

Even now, with Bortner almost a year out of cancer treatment, Mochi still digs through the trash out of habit.

Today, Bortner provides support to others going through the same experience, serving as a source of information about what to expect and how to keep going.

"It just brings a smile to my face when things come full circle — and now I'm on the other end, mentoring people through their cancer journeys," she said.

Dr. Marc Siegel, clinical professor of medicine at NYU Langone Health and Fox News senior medical analyst, confirmed that dogs have an "exquisite sense of smell."

"There is evidence in the medical literature that they can in fact smell the abnormal cancer proteins," the doctor told Fox News Digital. 

"In fact, artificial canine noses have been created relying on this response. Artificial intelligence is now enhancing this ability."

Pashtoon Kasi, M.D., medical director of gastrointestinal medical oncology at City of Hope in Orange County, California, was also not surprised by the idea that dogs could detect molecules or byproducts from cancer. 

"There is a lot of information that can be non-invasively found in blood, urine or other bodily fluids — or even the breath of individuals who have cancer in their bodies," he told Fox News Digital. 

"It has been demonstrated that dogs, and potentially other animals, are likely able to detect some of the odors associated with certain chemicals linked to cancer, which are detected in the blood or bodily fluids of patients with cancer." 

https://www.foxnews.com/health/woman-says-dog-detected-breast-cancer-before-doctors-known-whole-time

Lifesaving ChatGPT

A mother of two credits ChatGPT for saving her life, claiming the artificial intelligence chatbot flagged the condition leading to her cancer when doctors missed it.

Lauren Bannon, who divides her time between North Carolina and the U.S. Virgin Islands, first noticed in Feb. 2024 that she was having trouble bending her fingers in the morning and evening, as reported by Kennedy News and Media.

After four months, the 40-year-old was told by doctors that she had rheumatoid arthritis, despite testing negative for the condition.

Bannon, who owns a marketing company, then began experiencing excruciating stomach pains and lost 14 pounds in just a month, which doctors blamed on acid reflux.

Desperate to pinpoint the cause of her symptoms, Bannon turned to ChatGPT, the large-language model made by OpenAI.

The chatbot told Bannon that she may have Hashimoto's disease, an autoimmune condition where the body's immune system mistakenly attacks the thyroid gland, causing it to become inflamed and eventually underactive, according to Kennedy News and Media.

Despite reservations from her doctor, Bannon insisted on being tested for the condition in Sept. 2024 — and was shocked to discover that ChatGPT was correct, despite the absence of any family history.

This prompted doctors to perform an ultrasound of her thyroid, when they discovered two small lumps in her neck that were confirmed as cancer in Oct. 2024.

"I needed to find out what was happening to me. I just felt so desperate. I just wasn't getting the answers I needed."

Bannon claimed she would never have found the hidden cancer without the help of ChatGPT, which she credits for helping to save her life.

"I felt let down by doctors," said Bannon, as Kennedy News and Media reported. "It was almost like they were just trying to give out medication for anything to get you in and out the door."

"I needed to find out what was happening to me," she also said. "I just felt so desperate. I just wasn't getting the answers I needed."

Bannon said she had been using ChatGPT for work. When she asked the chatbot about which medical conditions mimic rheumatoid arthritis, it answered, "You may have Hashimoto's disease, ask your doctor to check your thyroid peroxidase antibody (TPO) levels."

After her cancer diagnosis in Jan. 2025, Bannon underwent an operation to remove her thyroid and two lymph nodes from her neck. She will remain under lifelong monitoring to ensure that the cancer doesn't return, according to the report.

Since she didn't present with typical symptoms of Hashimoto's disease, Bannon believes her condition, and her subsequent cancer diagnosis, would have remained undetected without the help of ChatGPT.

"I didn't have the typical symptoms of Hashimoto's disease — I wasn't tired or feeling exhausted," she said, per Kennedy News and Media.

"If I hadn't looked on ChatGPT, I would've just taken the rheumatoid arthritis medication and the cancer would've spread from my neck to everywhere else."

"It saved my life. I would've never discovered this without ChatGPT. All my tests were perfect."

Bannon is encouraging others to use the chatbot to investigate their own health concerns, but to "act with caution."

"If it gives you something to look into, ask your doctors to test you," she suggested. "It can't do any harm. I feel lucky to be alive."

Dr. Harvey Castro, a board-certified emergency medicine physician and national speaker on artificial intelligence based in Dallas, Texas, said he welcomes the role of AI tools like ChatGPT in raising awareness and prompting faster action, but also urges caution.

"AI is not a replacement for human medical expertise," Castro, who was not involved in Bannon's care, told Fox News Digital. "These tools can assist, alert and even comfort — but they can’t diagnose, examine or treat."

"When used responsibly, AI can enhance healthcare outcomes — but when used in isolation, it can be dangerous," the doctor went on. "We must prioritize patient safety and keep licensed medical professionals at the center of care."

https://www.foxnews.com/health/woman-says-chatgpt-saved-her-life-helping-detect-cancer-which-doctors-missed

Refusal to treat mother in labor

SAN DIEGO — As Hannah Michaelis groaned in agony in the front seat of her mother's car on the steps of Sharp Grossmont's Labor and Delivery Unit, her mother, Carla Michaelis, pleaded with a 911 dispatcher for help.

"My daughter's in the car, she's six months pregnant and is in excruciating pain. They said they can't see her here," said Carla Michaelis.

Michaelis had rushed her daughter to the closest hospital to her home. Michaelis rushed inside to let nurses know that her daughter, Hannah, needed immediate attention. 

Michaels says the nurses refused, telling Michaelis that she needed to drive her daughter to another hospital that specializes in extremely premature babies.

Unsure what to do to help her daughter, Michaelis called 911.

"Unfortunately, if you're at the hospital, we don't respond to the hospital to take you to a different hospital," said the dispatcher.

"They told me to take her to [UC San Diego], I can't take her to UCSD, she's in excruciating pain," Michaelis said. "She's screaming."

Carla Michaelis started driving to UCSD, but as she drove out of Sharp Grossmont, her daughter's cries intensified.

Michaelis turned her car around. She parked at the hospital doors and ran inside to beg for help.

When nurses finally agreed, Carla Michaelis said they put Hannah in a wheelchair, unbeknownst to them that Samuel was already born. The force of Hannah's weight on Samuel's head and body caused Samuel to suffer a catastrophic brain injury. 

Within five days, Samuel, Hannah's first baby and Carla's first grandbaby, was dead.

Now, nearly a year to the date, Hannah Michaelis and her mother, Carla, are suing Sharp Grossmont for medical negligence.

"Hannah and I want justice for our family," Carla told CBS 8. "We don't want this to ever happen to anybody else. We want whatever change needs to occur. No one should be turned away from the hospital like we were."

CBS 8 interviewed Carla Michaelis about the incident as her daughter, Hannah, was unable to conduct the interview.

Carla Michaelis was worried her daughter, Hanna, wouldn't get the type of medical care she needed for her first pregnancy. 

The 29-year-old pregnant mother had moved to rural Tennessee to be with her partner and the father of her baby, Samuel.

Carla Michaelis flew Hannah to San Diego in March 2024 to stay with her and get the medical care she needed.

"There's not really a lot of hospitals or doctors near where she was living," Carla Michaelis told CBS 8. "I begged her to come home so I could make sure she had really good care, and so she'd have, you know, good hospitals, good doctors at her disposal at any time that she needed. So I begged her to come home and let me take care of her through her pregnancy."

Hannah arrived in San Diego County in March 2024, four months pregnant and eager to become a mother. Her mother, Carla, was just as excited.

"Hannah was taking the utmost care of herself. I had made sure she had all the prenatal vitamins. She was eating healthier than she's ever eaten and taking better care of herself," said Carla Michaelis. "It was too important to her and to me for us to do anything other than take care of that body so we could have that baby."

On May 4, 2024, Carla Michaelis says Hannah asked to go to the hospital after feeling discomfort and seeing a small amount of discharge. It was the second time in a handful of days that Hannah had requested to see a doctor.

Carla Michaelis said she took Hannah to the nearest hospital, Sharp Grossmont, for observation. Michaelis said the doctors and nurses checked Hannah out and ran some tests, but didn't find anything out of sorts.

Carla Michaelis said doctors told her and Hannah to come back to Grossmont or go to another hospital if Hannah's condition worsened. 

The next day, May 5, it did.

"I woke up and Hannah was very uncomfortable," Carla Michaelis said. "She was yelling in agony. She told me she felt pressure, and at that moment, I told her to get her clothes on. I knew feeling pressure was not good."

Michaelis rushed her daughter to Sharp Grossmont. 

Familiar with the layout of the La Mesa hospital, and knowing her daughter was unable to walk, Michaelis drove to the Labor and Delivery Unit's front doors and rushed in for help as Hannah Michaelis screamed in pain inside the car.

Michaelis said she went to a group of nurses who were standing at the main desk.

"I went over to the lady closest to me, and I begged her. I said, 'I need your help. My daughter's in the car, and she needs your help.' I said that we were at the hospital the night before, and that we need your help."

The nurses, said Carla Michaelis, asked how far along Hannah was. Michaelis said 24 weeks.

"She said, 'We can't help you here,'" remembered Michaelis. "You're going to have to take her someplace else. And I said, 'No, you don't understand; I can't take her anywhere else. ' I was told to bring her back to the nearest emergency room, and I'm here. And she said, 'We can't help you here.'"

Michaelis walked outside to the car, where Hannah was now writhing in pain. 

Michaelis called 911 for help.

"Hello, I'm at Sharp Grossmont Hospital for Women and Newborns," Michaelis told the dispatcher, according to transcripts of the call obtained by CBS 8. "My daughter's in the car, she's six months pregnant, and she's having excruciating pain. They said they can't really see her here because if she is in labor, the baby...they can't take care of it. So they told me to take her to UCSD. I can't drive her to UCSD in this. She's in excruciating pain. She's screaming."

The dispatcher reported a problem. She couldn't dispatch an ambulance to a hospital.

"Unfortunately, the fire department, if you're at the hospital, we don't respond to the hospital to transfer you to a different one," the dispatcher told Michaelis. 

Carla Michaelis hung up the phone and began driving to UCSD to seek help for Hannah.

A few minutes later, the same 911 dispatcher called Sharp Grossmont to check on the situation.

"I just had a mother call in," said the dispatcher. "I guess she has her daughter. Are you guys with her or I don't know? She hung up talking."

The nurse responded, "Yes, I don't know she's yelling and screaming. I have a nurse going out to her car right now. She was told to go to UCSD yesterday if she had any further complications, and she just kind of stormed out, saying she was going to call 911, and I was like, No, they're not going to take you to another hospital."

Said the dispatcher, "Okay, so I just want to confirm you guys do not need the fire department. You will handle, correct?"

The nurse replied, "No, we do not need that. Thank you so much."

Meanwhile, Carla Michaelis drove just a few feet before turning around and heading back into Sharp Grossmont's Labor and Delivery Unit.

 "I blew past the front desk, went straight to the nurses who were standing there, and I said, 'My daughter's in pain. She needs you, and you're going to do your job, and you're going to come out and you're going to get that wheelchair, and you're going to come out to my car and you're going to help her right now," Carla Michaelis remembers saying.

When the nurses and Michaelis got back to the car, Hannah Michaelis was in pain. Her water broke. She told her mom that she thought she might have given birth already.

Despite the information, Carla Michaelis said the nurse refused to check Hannah and put her in a wheelchair and carted her off to the hospital.

Carla Michaelis tells CBS 8 that Hannah did, in fact, deliver the two-pound baby Samuel and that he became severely injured by not getting taken out of Hannah's pants before nurses transported her to the room. 

Nurses and doctors immediately rushed Baby Samuel to an incubator. He was badly bruised but moving and alert. 

Over the next five days, Hannah Michaelis, her partner Sam, and Carla Michaelis huddled together in the hospital, visiting Samuel as often as they could. 

However, the baby's condition worsened. After getting transported to UC San Diego Medical Center, Samuel developed a brain bleed. 

On May 9, after being told Samuel would not survive on his own, Hannah Michaelis and her partner agreed to remove Samuel from life support. 

"I rack my brain all the time thinking, what else could I have done?" asked Carla Michaelis. "Would she have been better staying in Tennessee, in rural Tennessee? Should I have taken her to a different hospital? I rack my brain all the time thinking what else could I have done, but I took her where I thought she was going to get the best care."

Attorney Janna Trolia represents Carla and Hannah Michaelis in a newly filed lawsuit against Sharp Hospital. Trolia says the 15 minutes when Hannah was sitting in pain inside her mother's car, instead of getting the help she needed, meant life and death for Samuel.

"If Hannah had been immediately triaged and brought into a labor and delivery suite or operating room—rather than left to give birth alone in her underwear outside the labor unit—Baby Samuel would have received life-saving interventions the moment he was born," said attorney Trolia.

"We find it profoundly disturbing that Grossmont staff mocked and ridiculed Carla as she pleaded for assistance, while Hannah was forced to deliver her firstborn child into her underwear just outside the hospital's entrance."

Trolia said the Emergency Medical Treatment and Labor Act (EMTALA) law requires hospitals to admit pregnant mothers regardless of their condition or how far along they are in their pregnancy.

Meanwhile, Carla Michaelis said she and her daughter are attending counseling together, but something is missing when she looks at Hannah.

"She doesn't want her business out, but she's not okay. She's healing, but there is irreparable harm. She's a completely different person."

CBS 8 reached out to Sharp Hospital for comment. A spokesperson said the hospital was unable to comment due to patient privacy laws and pending litigation.

https://www.cbs8.com/article/news/local/her-newborn-baby-died-after-a-hospital-refused-to-treat-her/509-6ccdaa51-6951-4bac-8f73-e6efbe20109a

Internal decapitation 4

An Illinois woman is detailing her remarkable and lengthy recovery journey after being internally decapitated in a life-changing accident.

Megan King was 16 years old when she fell to the ground while trying to jump and catch a soccer ball during her high school gym class. The accident, which occurred in 2005, left her with damage to her right ankle and spine. She also tore the muscle off both shoulder blades, according to the Daily Mail.

Per the outlet, King spent more than a year on crutches, but her condition got worse instead of better, causing her unbearable pain. Over the years, she underwent 22 surgeries but doctors were left stumped as she was unable to heal properly.

In 2015, doctors finally diagnosed King with Hypermobile Ehlers-Danlos syndrome (hEDS).

Ehlers-Danlos syndrome is a group of inherited disorders that affect your connective tissues — primarily your skin, joints and blood vessel walls, according to the Mayo Clinic. Symptoms include overly flexible joints and stretchy, fragile skin. The most common type of the disorder is hEDS, and people diagnosed with it often deal with joint pain and easy dislocations.

A year after her diagnosis, King was fitted for a halo brace to stabilize her neck and spine. However, while removing the brace, she was internally decapitated, per the Daily Mail. 

Atlanto-occipital dislocation (AOD) — also known as orthopedic decapitation or internal decapitation — occurs when ligaments and/or bony structures connecting the skull to the spine are damaged, according to a paper published in the World Journal of Orthopedics. 

“I flew my chair back to keep gravity from decapitating me,” King, now 35, told the Daily Mail. “My neurosurgeon had to hold my skull in place with his hands. I couldn't stand. My right side was shaking uncontrollably.”

AOD is typically fatal, with 90% of cases resulting in immediate death. However, doctors were able to rush King into emergency surgery and perform a spinal fusion to reconnect her skull and spine. The bones are fused together and heal as one.

 “It was a horror show,” she recalled, per the outlet. “I woke up unable to move my head at all.”

King was able to survive the harrowing ordeal, but has since had 37 surgeries in her recovery journey. The accident left her fused from her skull down to her pelvis, and she can no longer shift her head in any direction. “I'm literally a human statue,” she said. “My spine doesn't move at all. But that doesn't mean I've stopped living.”

According to the U.K. publication, doctors told King that her internal decapitation was likely due to a combination of her fall in high school as well as her weakened joints from living with hEDS.

Now, 20 years later, King is slowly but surely starting to return to her normal activities, boasting that she was able to go bowling recently. “I bowled a strike — on my very first try. My friends screamed and clapped and cheered like wild. They weren’t just celebrating the strike. They were celebrating everything I’ve survived,” she said, per the outlet.

King added, “I’m still learning what my new body can do. It’s not easy, but I’m adapting. And I’m always surprised by what I can still accomplish.”

https://people.com/decapitated-woman-is-a-human-statue-after-doctors-reattach-her-skull-11717350

A 12-year-old boy from Jerusalem, Israel is recovering after surgeons reattached his head following a bike accident that left him internally decapitated.

Suleiman Hassan’s “routine bike ride almost ended in disaster” when he “was forcefully run over by a wild driver," said the hospital in Jerusalem where the surgery took place — Hadassah Medical Center Ein Kerem — in a statement on their website.

“The injury left him with a fracture in the head and neck joint and tears in all his supporting ligaments,” they continued, adding that Hassan “was turned to Hadassa Ein Kerem by helicopter and was immediately admitted for a long and complex emergency surgery.”

“Due to the serious injury the head almost completely disconnected from the base of the neck," said Dr. Ohad Einav, a specialist orthopedist who performed the surgery on Hassan, in the statement.

Dr. Ziv Asa — another surgeon involved in the operation — said that there is typically a "50% chance of survival" for a case like Hassan’s. "Despite the serious injury — the surgery went very successfully, and Suliman was released to his home with a neck fixed and under close supervision of the hospital staff," the statement continued.

On Instagram, the hospital shared an image of Hassan with Einav and Asa following what they described in the post's caption as an "extremely rare and complex operation," where Hadassah Medical Center surgeons "reattached a 12-year-old boy’s head to his neck after a serious accident in which he was hit by a car while riding his bicycle."

The caption on the July 6 post continued, "Suleiman Hassan, from the Jordan Valley, was airlifted to Hadassah’s trauma unit in Ein Kerem, where it was determined that the ligaments holding the posterior base of his skull were severed from the top vertebrae of his spine. The condition, bilateral atlanto occipital joint dislocation, is commonly known as internal or orthopedic decapitation. The injury is very rare in adults, and even more so in children."

Atlanto-occipital dislocation (AOD), is also known as orthopedic decapitation or internal decapitation and occurs when ligaments and/or bony structures connecting the skull to the spine are damaged, according to a paper published in the World Journal of Orthopedics. It is a common cervical spine injury in motor vehicle accident deaths.

“We fought for the boy’s life,” Einav told The Times of Israel. “The procedure itself is very complicated and took several hours. While in the operating room, we used new plates and fixations in the damaged area… Our ability to save the child was thanks to our knowledge and the most innovative technology in the operating room.”

“The injury is extremely rare,” he added, “but we do know that because children between ages four and 10 have heads that are large in relation to their bodies, they are more susceptible than adults.”

The surgery was carried out in June, but the doctors waited a month to reveal the outcome, Fox News reported. Hassan was recently sent home from the hospital with a neck brace and will remain under medical supervision, according to the outlet.

"The fact that such a child has no neurological deficits or sensory or motor dysfunction and that he is functioning normally and walking without an aid after such a long process is no small thing," Einav said, according to the outlet.

Hassan’s father did not leave his son’s bedside during his recovery, the medical staff said, per The Times of Israel.

“I will thank you all my life for saving my dear only son,” the boy’s father said. “Bless you all. Thanks to you he regained his life even when the odds were low and the danger was obvious. What saved him were professionalism, technology and quick decision-making by the trauma and orthopedics team. All I can say is a big thank you.”

https://people.com/doctors-reattach-12-year-old-boy-s-head-after-bike-accident-7561123

CT scans and cancer

Smith-Bindman R, Chu PW, Azman Firdaus H, Stewart C, Malekhedayat M, Alber S, Bolch WE, Mahendra M, Berrington de González A, Miglioretti DL. Projected Lifetime Cancer Risks From Current Computed Tomography Imaging. JAMA Intern Med. 2025 Apr 14:e250505. doi: 10.1001/jamainternmed.2025.0505. Epub ahead of print. PMID: 40227719; PMCID: PMC11997853.

Abstract

Importance: Approximately 93 million computed tomography (CT) examinations are performed on 62 million patients annually in the United States, and ionizing radiation from CT is a known carcinogen.

Objective: To project the number of future lifetime cancers in the US population associated with CT imaging in 2023.

Design, setting, and participants: This risk model used a multicenter sample of CT examinations prospectively assembled between January 2018 and December 2020 from the University of California San Francisco International CT Dose Registry. Data analysis was conducted from October 2023 to October 2024.

Main outcomes and measures: Distributions of CT examinations and associated organ-specific radiation doses were estimated by patient age, sex, and CT category and scaled to the US population based on the number of examinations in 2023, quantified by the IMV national survey. Lifetime radiation-induced cancer incidence and 90% uncertainty limits (UL) were estimated by age, sex, and CT category using National Cancer Institute software based on the National Research Council's Biological Effects of Ionizing Radiation VII models and projected to the US population using scaled examination counts.

Results: An estimated 61 510 000 patients underwent 93 000 000 CT examinations in 2023, including 2 570 000 (4.2%) children, 58 940 000 (95.8%) adults, 32 600 000 (53.0%) female patients, and 28 910 000 (47.0%) male patients. Approximately 103 000 (90% UL, 96 400-109 500) radiation-induced cancers were projected to result from these examinations. Estimated radiation-induced cancer risks were higher in children and adolescents, yet higher CT utilization in adults accounted for most (93 000; 90% UL, 86 900-99 600 [91%]) radiation-induced cancers. The most common cancers were lung cancer (22 400 cases; 90% UL, 20 200-25 000 cases), colon cancer (8700 cases; 90% UL, 7800-9700 cases), leukemia (7900 cases; 90% UL, 6700-9500 cases), and bladder cancer (7100 cases, 90% UL, 6000-8500 cases) overall, while in female patients, breast was second most common (5700 cases; 90% UL, 5000-6500 cases). The largest number of cancers was projected to result from abdomen and pelvis CT in adults, reflecting 37 500 of 103 000 cancers (37%) and 30 million of 93 million CT examinations (32%), followed by chest CT (21 500 cancers [21%]; 20 million examinations [21%]). Estimates remained large over a variety of sensitivity analyses, which resulted in a range of 80 000 to 127 000 projected cancers across analyses.

Conclusions and relevance: This study found that at current utilization and radiation dose levels, CT examinations in 2023 were projected to result in approximately 103 000 future cancers over the course of the lifetime of exposed patients. If current practices persist, CT-associated cancer could eventually account for 5% of all new cancer diagnoses annually.

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CT scans, or CAT scans, are widely used to get internal images of the body and diagnose dangerous medical conditions — but they could pose a hidden risk.

A new study from the University of California - San Francisco found that CT (computed tomography) scans could be responsible for 5% of all cancer diagnoses each year, according to a press release.

"While some uncertainty exists, it doesn’t significantly affect the core conclusion: A small but meaningful percentage of cancers are linked to CT scans, and this number can be reduced," first author Rebecca Smith-Bindman, MD, a UCSF radiologist and professor, told Fox News Digital.

The researchers’ estimates show that the ionizing radiation exposure from CT scans is comparable to other significant risk factors, such as alcohol consumption and excess body weight.

To predict how many future cancers could result from current CT scans, the researchers updated a previous analysis of 2023 data on scan volumes, scan types and radiation doses, according to Smith-Bindman.

"We used a well-validated model to estimate cancer risk and conducted sensitivity analyses to confirm the robustness of our findings," she said.

"This is a modeling study, meaning our conclusions depend on the accuracy of the data used."

Based on the review, the researchers estimate that approximately 103,000 future cancers will be caused by CT scans performed in 2023 in the U.S., with the highest number of cases affecting adults aged 50 to 69.

Individual cancer risk is highest for babies and children, with babies under 1 year old 10 times more likely to develop the disease compared to others in the study.

Adults, however, account for the majority of scans, which drives the overall cancer burden, Smith-Bindman noted.

"CT doses are sometimes higher than necessary."

The most common cancer types resulting from CT radiation, according to the study, include lung cancer, colon cancer, leukemia and breast cancer.

The study, which was published on April 14 in JAMA Internal Medicine, received funding from the National Institutes of Health.

"In many cases, CT is the most appropriate test for achieving rapid and accurate diagnoses," Smith-Bindman told Fox News Digital.

"However, the use of CT continues to rise, including a concerning increase in imaging that is performed without a justified medical reason — often referred to as ‘low-value scanning.’"

Based on the findings, the researchers recommend avoiding unnecessary CT scans to avoid potential harms.

Another risk-reducing approach is to lower the radiation dose per scan.

"CT doses are sometimes higher than necessary, so patients are encouraged to ask their healthcare providers or technologists to use the lowest possible dose for their scan," Smith-Bindman advised.

Ultimately, the researcher said, patients should have informed conversations with their healthcare providers about the necessity of a CT scan and whether alternative imaging options — like ultrasound or MRI — might be more appropriate.

"If CT is clearly indicated, the benefits far outweigh the risks," she said. "But if not, it’s best to avoid the scan altogether."

"Less can be more when it comes to good patient care."

Dr. Nicole Saphier, board-certified radiologist and Fox News medical contributor, was not involved in the study but commented on the risks and benefits of CT scans.

"I have long advocated for the judicious use of medical imaging, often citing that less can be more when it comes to good patient care," she told Fox News Digital.

"The recent study linking the ionizing radiation from CT scans to an increased risk of cancer underscores what many in the medical community have understood for years: while imaging is a powerful diagnostic tool, it is not without risk."

Saphier said the study may even underestimate the total number of cancers attributable to medical imaging.

"Many cases of medical intervention-induced cancers may go unrecognized due to long latency periods, the complex interplay of contributing factors and the fact they omitted radiation from image-guided procedures, x-rays and other forms of medical radiation from this study," she noted.

"As imaging use and medical interventions continue to rise — especially in younger populations — the cumulative radiation exposure over a lifetime becomes an increasingly important consideration."

That said, Saphier went on, CT scans and other medical imaging modalities — which are "fast and cheap" — save lives every day.

"The key is balance. Physicians must remain vigilant in weighing the benefits against the risks, and should always explore alternative modalities when appropriate, such as ultrasound or MRI, which do not use ionizing radiation," she said.

Informed decision-making is essential for both clinicians and patients, according to the doctor.

"We must continue to refine our protocols, limit unnecessary imaging and ensure we are using the lowest possible doses without compromising diagnostic quality," Saphier concluded.

"This is not a call to avoid CT scans — it is a call to use them wisely."

https://www.foxnews.com/health/common-medical-test-linked-cancers-study-suggests-use-them-wisely

Fremanezumab for prevention of episodic migraine in children and adolescents

Fremanezumab (Ajovy)—a calcitonin gene-related peptide agonist (CGRP)— is safe and effective for prevention of episodic migraine in children and adolescents as young as 6 years old, according to findings presented at the AAN 2025 Annual Meeting in San Diego.

The multicenter, double-blind, parallel-group, phase 3 study—led by headache medicine specialist and child neurologist Andrew Hersey, MD, PhD, FAAN, endowed chair and director of the division of neurology at Cincinnati Children's Hospital Medical Center—randomized participants aged 6–17 years, with a migraine diagnosis for ≥6 months and history of ≤14 headache days/month, 1:1 to monthly fremanezumab (<45kg, 120 mg; ≥45kg, 225 mg) or placebo for 12 weeks.

“The primary endpoint was a mean change from baseline in average monthly migraine days (MMD), with secondary endpoints including mean change from baseline in monthly headache days of at least moderate severity (MHD) and proportion of participants achieving a ≥50% reduction in MMD," the investigators wrote in their abstract.

Over three months, fremanezumab significantly reduced MMD vs. placebo (-2.5 vs -1.4, p=0.0210). The reduction in MHD was significantly greater with fremanezumab versus placebo (–2.6 vs –1.5; p=0.0172), as was the 50 percent response rate (47.2 percent vs 27percent (p=0.0016).

The trial also included subgroup analyses conducted by age (6–11 and 12–17 years) and sex. In all subgroups, mean changes from baseline in MMD favored fremanezumab over placebo: age (6–11 years: – 3.4 vs –1.7; 12–17 years: –2.7 vs –1.8) and sex (male: –3.5 vs –2.2; female: –2.3 vs –1.5).

The proportion of participants reporting more than one adverse event was similar across treatment groups and the proportion of participants with serious adverse events (less than 3 percent) and adverse events leading to discontinuation were low at less than 1.0 percent.

“These findings demonstrate the efficacy, safety, and tolerability of fremanezumab in children and adolescents with episodic migraine," the investigators concluded.

“This is extremely significant," said Jessica Ailani, MD, clinical professor of neurology at MedStar Georgetown University Hospital and director of the MedStar Georgetown Headache Center in Washington, DC. “This is the first readout of study results for any of the anti-CGRP treatments for migraine prevention in the pediatric population, and as a field we've been holding our breath hoping for an effective treatment. This data is positive, and I'm extremely encouraged."

“While we have real-world evidence showing that these anti-CGRP treatments, especially the monoclonal antibodies, are effective in the pediatric population, that real-world data has been focused more on patients who are refractory to other treatments," Dr. Ailani said. “So it is useful to have trial data in groups of patients who aren't necessarily refractory, and in an age range that includes fairly young patients."

She also praised the trial's breakdown by sex. “There has been some controversy about whether anti-CGRP preventive treatments are equally effective in women and men," she said. “They broke that down, and it looked like maybe it was about a quarter of a day more effective in the male population than the female, but overall, it looked to be very effective in both sexes."

Dr. Ailani noted that in the pediatric population, especially with injectables, there are often a lot of dropouts because of adverse events, so the low dropout rate was impressive. “But I'd like to see if the actual adverse event profile resembled the adult studies, where we didn't really see much other than injection site reactions."

A headache therapy outperforming placebo in the pediatric population is a significant achievement, Dr. Ailani said. “In migraine, we have really struggled to show efficacy compared to placebo, even for acute treatments that we know work really well," she said.

“If we get these patients on track early with migraine-specific treatments, is it possible that they can grow up and not develop chronic migraine?" she asked. “I would love to see long-term data on these types of treatments, and what the headache freedom response rates look like over the course of a year for patients who remain on treatment. Could we see rates of chronic migraine go down over time? That's a really exciting possibility."

Disclosures: Dr. Hershey has nothing to disclose. Dr. Ailani received consulting fees from Abbvie, Eli Lilly, Lundbeck, Satsuma, Pfizer, Gore, Ipsen, Merz, Scilex Dr. Reddy, Linpharma, Vectura, and Aeon. Dr. Ailani serves on a scientific advisory or data safety monitoring board for Abbvie, Linpharma, and Aeon. Dr. Ailani's institution has received research support from Ipsen, Parema, and Lundbeck.

Efficacy and Safety of Fremanezumab for the Preventive Treatment of Episodic Migraine in Children and Adolescents: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study
Andrew D. Hershey1, Christina L. Szperka2, Piero Barbanti3, Patricia Pozo-Rosich4, Petra Bittigau5, Steve Barash6, Sally Garnett6, Juline Bryson6, Yoel Kessler7, Yael Carmeli Schwartz7, Xiaoping Ning6
1Cincinnati Children’s Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, OH, USA, 2Children’s Hospital of Philadelphia and Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA, 3Headache and Pain Unit, IRCCS San Raffaele, Rome, Italy, and San Raffaele University, Rome, Italy, 4Headache Unit and Research Group, Vall d’Hebron Hospital and Research Institute, Universitat Autonoma de Barcelona, Barcelona, Spain, 5Charité-Universitätsmedizin Berlin, Berlin, Germany, 6Teva Branded Pharmaceutical Products R&D, Inc., West Chester, PA, USA, 7Teva Pharmaceutical Industries Ltd., Tel Aviv, Israel

Objective:

The SPACE study (NCT04458857) evaluated the efficacy, safety, and tolerability of fremanezumab in children and adolescents with episodic migraine (EM).

Background:

Fremanezumab is a calcitonin gene-related peptide pathway monoclonal antibody approved for preventive migraine treatment in adults.

Design/Methods:

In this multicenter, double-blind, parallel-group, Phase 3 study, participants aged 6–17 years, with a migraine diagnosis for ≥6 months and history of ≤14 headache days/month, were randomized (1:1) to monthly fremanezumab (<45kg, 120 mg; ≥45kg, 225 mg) or placebo for 12 weeks. Primary endpoint: least-squares (LS) mean change from baseline in average monthly migraine days (MMD) during the double-blind period. Secondary endpoints included LS mean change from baseline in monthly headache days of at least moderate severity (MHD) and proportion of participants achieving a ≥50% reduction in MMD. Subgroup analyses were conducted by age (6–11 and 12–17 years) and sex.

Results:

Of 237 randomized participants, 234 (6–11 years, n=63; 12–17 years, n=171; male, n=105; female, n=129) were included in the efficacy analysis (fremanezumab, n=123; placebo, n=111). Fremanezumab significantly reduced MMD versus placebo (–2.5 vs –1.4; p=0.0210) over 3 months. LS mean changes from baseline in MMD favored fremanezumab over placebo in subgroups stratified by age (6–11 years: –3.4 vs –1.7; 12–17 years: –2.7 vs –1.8) and sex (male: –3.5 vs –2.2; female: –2.3 vs –1.5). The reduction in MHD was significantly greater with fremanezumab versus placebo (–2.6 vs –1.5; p=0.0172), as was the 50% response rate (47.2% vs 27.0%; p=0.0016). The proportion of participants reporting ≥1 adverse event (AE) was similar across treatment groups (fremanezumab, 55%; placebo, 49%). The proportion of participants with serious AEs (≤3%) and AEs leading to discontinuation (<1%) were low.

Conclusions:

These findings demonstrate the efficacy, safety, and tolerability of fremanezumab in children and adolescents with EM.

Children with peri-optic neuritis treated with intravenous steroids followed by oral steroids

Children with peri-optic neuritis, a rare disorder involving inflammation of the sheath surrounding the optic nerve, had generally favorable outcomes after treatment with intravenous steroids followed by oral steroids, according to a single-center case series presented at the AAN Annual Meeting in San Diego.

“We had both a neuro ophthalmologist and a neuroradiologist who were noticing that more cases of peri-optic neuritis seemed to be occurring at our institution," said the lead author Nicholas Benjamin, MD, a neurology fellow at the University of Pittsburgh.

“We looked into the literature and found very little in pediatric cases specifically," he told Neurology Today At the Meetings. “We decided to search for all the cases [occurring between January 2017 and March 2024] we could identify within our center."

To find potential cases of pediatric peri-optic neuritis, Dr. Benjamin and colleagues searched the ICD-10 codes for diagnoses including perineuritis, optic neuritis (ON), optic papillitis, retrobulbar neuritis, other ON and unspecified ON. For all cases, they reviewed documented interpretations of MRI brain/orbit imaging by radiology. They then reviewed imaging with a neuroradiologist to confirm the diagnosis.

“We began by looking at all patients with peri-neuritis on imaging and then went backwards to look at treatment they received," Dr. Benjamin said. “Many of these cases also demonstrated optic neuritis as well on imaging concurrently, so they were dual treated for these, as the primary teams were more often treating the optic neuritis picture, as they are more familiar with this etiology. As a result, a number of our reviewed cases did not mention peri-neuritis on imaging reads, but upon our look, clearly had peri-neuritis present."

Dr. Benjamin and colleagues ultimately identified 20 cases, nine of whom were females, with a median age at diagnosis of nine years.

Seventy five percent of the patients presented with changes in vision, headaches (45 percent), or painful eye movements (40 percent).

The most common diagnoses after review of imaging were idiopathic ON (in 35 percent), myelin oligodendrocyte glycoprotein (MOG)+ ON (in 35 percent), and inflammatory P-ON without ON (in 10 percent).

None of the patients was diagnosed with either multiple sclerosis of NMO. “I found that a little surprising and unexpected," Dr. Benjamin said. Seventeen of the 20 patients were tested for myelin oligodendrocyte glycoprotein antibody disease (MOGAD), seven of whom (41 percent) were positive.

Initial treatment for 18 of the patients was intravenous steroids, followed by oral steroids.

“Adults with isolated peri-optic neuritis can sometimes get away with receiving only oral steroids," Dr. Benjamin said. “Our findings suggest that kids seem to do well when they receive IV steroids followed by an oral course." However, many cases in this study had concurrent ON and perineuritis present at the same time, and received treatment for both of these conditions.

Only two of the patients required therapeutic escalation to plasmapheresis or the immunosuppressant drug rituximab.

Four patients experienced documented relapses. At follow-up, ranging from three to 10 months, all 16 patients with available data had stable or improved visual acuity.

“This is just a case series, “but it shows that IV steroids followed by oral steroids may be a good regimen for the pediatric population," Dr. Benjamin said.

Stronger evidence for the best treatment options would require a randomized trial, but doing so would be unlikely for a single institution due to the condition's rarity, Dr. Benjamin said.

“It could be done, but it would likely take multiple institutions collaborating," he said.

A neuroimmunologist familiar with the study said she was glad to see its focus on pediatric cases but hopes that larger studies will eventually be available.

“It's a small case series from a single site," said Jennifer Olson Graves, MD, PhD, professor of neurosciences and division chief of neuroimmunology at the University of California, San Diego, as well as director of the Rady Children's Pediatric MS Center in San Diego.

“Consistent with the adult literature, there were no MS cases with perineuritis," she said. “I teach people that if you see peri-optic neuritis, it's never MS."

She was surprised that only seven of the 17 cases tested for MOGAD were positive, and that only one-third of the cases were positive for MOG.

“We need to think of this as a frequency series in a tertiary care hospital, not a population-based study," Dr. Graves said. “It may be referral bias that most of their cases weren't MOG. I would be curious to know whether the ones who had to escalate were MOG positive or not. I would expect the MOG cases to be responsive to steroids."

Dr. Graves said that in her experience, “kids with MOGAD have about a 50 percent chance of relapse. For the idiopathic cases, it's hard to predict. It's possible that it would be a one-time event and never recur."​

Disclosures: Dr. Benjamin had no disclosures.​

https://journals.lww.com/neurotodayonline/blog/NeurologyTodayConferenceReportersAANAnnualMeeting/pages/post.aspx?PostID=213

Children with Peri-Optic Neuritis Have Favorable Visual Outcomes After Steroid Treatment: A Singe-Center Pediatric Case Series
Nicholas Benjamin1, Srikala Narayanan2, Catalina Cleves1, Levi Shelton1
1Children's Hospital of Pittsburgh of UPMC, 2Texas Children’s Hospital

Objective:

To determine baseline characteristics and outcomes in pediatric patients presenting with peri-optic neuritis (P-ON).

Background:

P-ON is a rare inflammatory disorder leading to inflammation of the nerve sheath. Most literature addresses adults, with little information on the pediatric population.

Design/Methods:

We conducted a single-center retrospective study of patients presenting to UPMC Children’s Hospital from 01/2017 – 03/2024. We used ICD-10 codes (optic neuritis (ON), optic papillitis, retrobulbar neuritis, other ON, unspecified ON) to identify potential patients with P-ON. For identified patients, we reviewed documented interpretations of MRI brain/orbit imaging by radiology. For patients with interpretations suspicious for P-ON, we reviewed their imaging with a neuroradiologist to confirm the diagnosis. For those patients, we collected baseline characteristics and outcomes.

Results:

We identified 20 cases of P-ON. The median age at diagnosis was 9 years, and 9 patients (45%) were female. Most patients presented with vision changes (75%), headaches (45%), and painful eye movements (40%). Initial ophthalmologic findings included disc edema (55%) and disc pallor (30%). The most common diagnoses (all with confirmed P-ON on imaging) were idiopathic ON (35%), MOG+ ON (35%), and inflammatory P-ON without ON (10%). No patients were diagnosed with MS or NMO. Of the 17 patients tested for MOGAD, 7 (41%) were positive. Eighteen (90%) patients initially received intravenous (IV) steroids, followed by oral steroids. Two (10%) patients had escalation of therapies to plasmapheresis or Rituximab. Four (20%) patients had documented relapses. Steroid therapy produced stable or improved visual acuity at follow-up (ranging from 3-10 months) in all patients (100%) with data present (n=16).

Conclusions:

MOG+ status was common among patients with P-ON, along with ON cases. Regarding visual outcomes, all patients either had stable or improved visual acuity at follow-up, showing that the regimen of IV to oral steroids may be a good treatment option in the pediatric population.

Alobar holoprosencephaly redux

 Anonymous donor paid $47K for baby's medical bills, family says

A Florida baby who was given just weeks to live is thriving today — and it wouldn’t have been possible without the generosity of an anonymous donor who covered her medical bills.

When Bill and Meg Longhenry welcomed their second child, Millie, in August 2023, they were told she had no hope of survival due to a rare and severe congenital brain disorder called alobar holoprosencephaly (HPE).

HPE affects about one in 10,000 live births, and most infants do not survive beyond the first week, statistics show. Millie was born with the most severe form of the disease.

"We found out that she has a rare brain malformation where part of her brain didn't develop, and the other part didn't develop correctly," Meg Longhenry said in an on-camera interview with Fox News Digital. 

"So there's no division between the two hemispheres and the middle is hollow."

Doctors told the parents that "Millie should have been a miscarriage or a stillbirth," her mother said. "She should have died moments after birth."

"They told us over 95% of patients with this diagnosis don't survive past the first few months … and anyone who survives past that requires an enormous deal of medical care, like feeding tubes and breathing tubes," said Bill Longhenry. "Usually they have no brain function."

After spending two months in the hospital, Millie was sent home on hospice care with four to six months to live — but the Longhenrys weren’t ready to give up.

"God had something else in mind," said Bill Longhenry. "God had a different plan, and only God was able to really make that decision."

"Millie should have been a miscarriage or a stillbirth," doctors told the baby’s parents.

A friend recommended that Millie’s parents connect with Dr. Brandon Crawford, a functional neurologist at the NeuroSolution Center of Austin, who specializes in using non-invasive techniques without drugs or surgery.

Upon reviewing MRIs and examining Millie, Crawford said he saw "huge potential."

While much of her brain is missing, he said, the higher portion is "relatively intact and functioning well," he told Fox News Digital.

"I started to get the idea that this kiddo is really trying — she's not on the decline, she's actually really fighting to live her life in this world."

Defying the odds

Under Crawford’s care, Millie began a treatment plan that included laser light therapies, acoustic wave therapy that uses sound waves to stimulate natural healing processes, and primitive reflex integration, which "retrains" the brain-body connection and helps babies learn to better control their movements.

Dr. Marcella Madera, a neurosurgeon who serves as NeuroSolution’s medical director, also collaborates on Millie’s treatment to ensure safety and efficacy.

"It’s this combination of regenerative medicine, developmental functional neurology, and photobiomodulation that’s sparking and fueling her brain development and building neuroplasticity," Crawford told Fox News Digital in a separate interview. 

"For example, she can clearly see and she responds to visual cues — yet she doesn't have the majority of those visual pathways developed in her brain," he went on. "That means her brain has rewired and remapped the ability to see, and that's the amazing part, that the brain is able to do that."

Bill Longhenry describes the treatment as "combining physical therapy with neural functions."

Today, Crawford said, Millie is not only surviving, but thriving — something that is very rare for this condition.

"She continues to grow and develop and is getting stronger," he said. "We're working on crawling with her right now — that's unheard of for this. Her joint attention continues to improve, even her ability to eat."

Millie is also starting to vocalize, Crawford said, saying "Mom" and "Dad" and communicating with her big brother, Theo.

"She's got a spunky little personality, and it's amazing," he said. "Honestly, if you look at her and interact with her in person and then look at her MRI, you wouldn't think it's the same kid."

Millie is smiling, laughing and responding to her name. She also understands people’s speech and is using sign language. 

"Millie would not be here today if we weren't doing the different things to help her brain, to help her rewire," added Meg Longhenry.

Answered prayers

Last month, Millie’s family faced the possibility of canceling her intensive neurological therapy due to financial constraints.

Meg Longhenry had recently let Crawford know they would have to cancel their next treatment due to lack of funds — but he told her to come in anyway.

"I said, don't worry about it, just come. There's no way I'm dropping care with Millie — we've come too far."

On the morning of March 27, as Crawford’s team was about to perform another regenerative medicine procedure with Millie, they prayed for divine intervention, he told Fox News Digital.

"A couple of hours later, we got the random phone call," he said. "It was another patient who has been following Millie's story, and she said, ‘I feel like I'm supposed to donate something for Millie’s case, and my front desk said, well, that would be amazing."

The donor offered to cover the total outstanding balance for Millie’s treatment — more than $47,000.

"It's just impossible to understand that level of generosity from a stranger," said Bill Longhenry. 

"We have to pursue this treatment, but it's not covered by insurance, so we're just doing whatever we can to make it work."

Ultimately, the Longhenrys did find out who covered the medical expenses — a previous patient of Dr Crawford’s. They were able to call her and thank her for the donation.

While this anonymous gift clears a major financial hurdle, Millie’s journey is far from over, the family shared. 

She will require follow-up therapy every four to six months, specialized home equipment and travel for continued care, which insurance does not cover.

"I think the finances are always really scary for us … but there's not a price that I could put on her life," Meg Longhery said. "I'll continue to fight and do what I need to do so she can have the best life that she can."

"There's not a price that I could put on her life."

The family also relies heavily on their faith, believing that Jesus worked through Dr. Crawford to help save Millie’s life, according to her mother.

"We serve such a big God that he is greater than our biggest fears — he is the greatest physician, and he aligns us with where we need to be and who we need to be," she said.

"And it's so encouraging to see the growth that we were told repeatedly we wouldn't see."

For more information about Millie’s journey and progress, people can visit MovingMountainsForMillie.org or @movingmountainsformillie on Instagram.

https://www.foxnews.com/health/baby-fatal-brain-disorder-saved-anonymous-47k-donation

Pelletier/Gottesfeld updates

Justina Pelletier is Focusing on Her Personal Growth Today

Once Justina returned home, she underwent a few more surgical procedures to help cope with her symptoms. Her parents also consulted doctors regarding potential spine-related problems which have hampered her walking. In 2020, Justina testified in court for a lawsuit her parents had filed against the BCH for medical malpractice. She alleged that the hospital staff disallowed her to speak to her parents and were sometimes cruel to her. Justina further insisted that her pleas to return home were ignored. She said, “They didn’t believe [my pain], and they hurt me so much,” Pelletier said. “I kept getting weaker.”

Since her discharge, Justina has engaged in art therapy and horseriding and graduated high school in 2020. She now still leads a private life in her parents’ home in Connecticut, where she feels happy and safe in their constant care. Now 23, Justina continues receiving treatment for her mitochondrial syndrome. Sadly, in 2021, she experienced a stroke, drastically impacting her health, verbal communication, and energy levels.

https://thecinemaholic.com/where-is-survivor-justina-pelletier-now/

Lou and Linda Pelletier Are Focusing on Justina’s Health Now

Though the Pelletiers gained significant support from religious groups, parent groups, and media outlets, Justina was shifted to Wayside Youth and Family Support Network, a residential facility in Framingham, Massachusetts. Seeing no other way to get back their daughter, Linda and Lou mildly complied with the BCH’s treatment plan for her, and in June 2014, the court finally dismissed the child protection case against them. Thus, Justina was finally reunited with them and taken back to Connecticut.

Linda and Lou were relieved to bring Justina home, but their troubles were far from over. The teenager was unable to walk and was still having multiple health complications. Deeply hurt over the trauma they and Justina had faced for around 16 months, Linda and Lou filed a malpractice lawsuit against Boston Children’s Hospital for sending her to the psychiatric ward. The couple’s testimony divulged how their daughter secretly passed them notes during their hospital visits, complaining of the staff’s misbehavior and her desperate wish to go home.

Not just that, Justina herself testified the same in court in 2020, but ultimately, the jury found BCH not negligent in her care. Despite losing the lawsuit, Lou refused to give up his fight for his daughter and helped pass the bill for Justina’s Law. It prohibits the use of federal funds to conduct or support treatment or research involving a ward of the state in which the individual’s health is subjected to greater than minimal risk with no or minimal prospect of direct benefit.

Lou and Linda still live in their Connecticut home and are dedicated to caring for Justina round-the-clock, as she is back on her medications for mitochondrial disease. The Pelletiers faced another setback in 2021 when she had a stroke, affecting her daily functions. Nevertheless, her parents took every measure to nurse her back to health and now hope she can walk again someday. Linda shared how traumatized she still feels about everything and how hard it is for her and Lou to put what happened behind them. We sincerely hope the family gets the strength to heal from their pain and have a peaceful future ahead.

https://thecinemaholic.com/lou-and-linda-pelletier-where-are-justinas-parents-now/

Marty Gottesfeld is Now Making the Most of His Time As a Free Man

Marty was sentenced to ten years in prison and was subsequently incarcerated in a few different federal prisons across the nation. According to his wife, there was even a time a high-security facility kept him in solitary confinement for 22 hours daily, and he was allowed only two fifteen-minute calls weekly. Dana divulged in the documentary that Marty’s communication were heavily monitored. Yet, he continues to support social causes and voices his opinions on government policies by corresponding with media organizations through her.

As per the documentary, the hacktivist’s projected release date was April 11, 2024, and he already had a job offer from a progressive publishing house to work as a web developer. However, he was released early, around November 2023, and has since happily reunited with his loving wife Dana. “Words fail me, because you’re out in public again,” he once said upon being asked what it was like to be free. “Like, they just put you on a greyhound bus; when I was released, it’s like, they just drop you off at the bus station, and you’re out in public again, and you can talk to people.” We should mention he did walk away with 210 lbs of legal documents he’d created while incarcerated, and he is determined to continue down the path he once created to help Justina and others like the once-teen. The Somerville, Massachusetts, resident is now a full-fledged human rights activist as well as a writer for many platforms; plus he has a blog of his own.

https://thecinemaholic.com/marty-gottesfeld-where-is-hacker-and-social-activist-today/

Schizophrenia is associated with an aberrant immune response to Epstein–Barr virus

Faith Dickerson, Lorraine Jones-Brando, Glen Ford, Giulio Genovese, Cassie Stallings, Andrea Origoni, Colm O’Dushlaine, Emily Katsafanas, Kevin Sweeney, Sunil Khushalani, Robert Yolken, Schizophrenia is Associated With an Aberrant Immune Response to Epstein–Barr Virus, Schizophrenia Bulletin, Volume 45, Issue 5, September 2019, Pages 1112–1119, https://doi.org/10.1093/schbul/sby16

Abstract

Background

Epstein–Barr virus (EBV) is a highly prevalent human herpesvirus capable of infecting the central nervous system and establishing persistent infection.

Methods

We employed solid phase immunoassay techniques to measure immunoglobulin G (IgG) class antibodies to EBV virions and defined proteins in 432 individuals with schizophrenia and 311 individuals without a history of a psychiatric disorder. Western blot testing was performed to document reactivity to specific EBV proteins. Polygenic risk for schizophrenia was calculated from genome sequencing arrays. Levels of antibodies between the groups were compared by multivariate analyses incorporating clinical, genetic, and demographic measures.

Results

Individuals with schizophrenia had marked elevations in the levels of antibodies to EBV virions as compared to the control population. Further analyses indicated increased levels of reactivity to EBV-viral capsid antibody (VCA) but not to EBV nuclear antigen-1 (EBNA-1) or to other human herpesviruses. Western blot analysis confirmed increased reactivity to VCA proteins in the group of individuals with schizophrenia and documented a lack of increased levels of antibodies to EBNA-1. Genetic analyses indicated an additive effect of increased levels of antibodies to EBV virions and genetic susceptibility to schizophrenia, with individuals with elevated levels of both type of markers having a greater than 8.5-fold odds of a schizophrenia diagnosis.

Conclusions

Individuals with schizophrenia have increased levels of antibodies to some but not all EBV proteins indicating an aberrant response to EBV infection. This aberrant response may contribute to the immunopathology of schizophrenia and related disorders.
_____________________________________________________


New research from Johns Hopkins Medicine and Sheppard Pratt Health System shows that people in the study with schizophrenia also have higher levels of antibodies against the Epstein-Barr virus (EBV), a herpes virus that causes infectious mononucleosis, so-called mono.

Researchers proposed two explanations for the association of heightened immune responses in patients with schizophrenia and EBV infection: schizophrenia might alter the immune systems of these patients and make them more susceptible to EBV, or EBV infection might increase the risk of schizophrenia.

The article was published online Nov. 20 in Schizophrenia Bulletin.

“We are interested in the role of infectious agents such as Epstein-Barr virus in schizophrenia and other serious psychiatric disorders, so we did this study to look at the associations,” said Robert Yolken, M.D., the Theodore and Vada Stanley Distinguished Professor of Neurovirology in pediatrics at Johns Hopkins Children’s Center and senior author of the study. Yolken cautioned that the study wasn’t designed to determine cause and effect.

Schizophrenia is a mental disorder where patients have distorted thinking, perception, emotions, language, sense of self and behavior. According to the World Health Organization, schizophrenia affects more than 21 million people worldwide.

While schizophrenia has some genetic associations, genes that have been found to date explain only a portion of the disease risk. Environmental exposures, including to some infectious agents, have also been identified in previous studies as increasing the risk for schizophrenia.

EBV is a common virus and research suggests it infect approximately 90-95 percent of the world’s population. However, most people never show symptoms of infection and the vast majority of people never have complications. EBV initially causes fever and swollen lymph nodes, and is commonly transmitted through oral contact such as kissing. In severe cases, it can spread to the central nervous system and cause persistent infection. Researchers wanted to see the relationship between this EBV infection and schizophrenia.

The researchers conducted a study among 743 people—432 with a schizophrenia diagnosis and 311 without a history of a psychiatric disorder to serve as a control group. Around 55 percent of the participants were men.

The researchers first measured levels of antibodies against components of EBV by comparing antibody levels in healthy people with those of people who have schizophrenia. They looked at the odds of having these antibodies in the 50th, 75th and 90th percentiles and found that people with schizophrenia were 1.7 to 2.3 times more likely to have increased levels of some EBV antibodies compared with people without schizophrenia.

Then they measured the antibodies to other related viruses such as varicella/chicken pox or herpes simplex type 1/cold sore virus, and didn’t find an increase of antibodies against these viruses in people with schizophrenia. These findings suggest that only EBV was associated with increased risk of schizophrenia.

After that, the researchers sequenced a portion of the participants’ DNA to determine their genetic risk for schizophrenia. Results from the analysis showed that people who had both evidence of increased genetic risk for schizophrenia and increased antibody levels to EBV had a more than eight times higher chance of being in the schizophrenia group as compared with controls. Approximately 10 percent of the individuals with schizophrenia had increased levels of both antibodies and genetic risk as compared with slightly more than 1 percent of the controls.

“We found that individuals with schizophrenia had an unusual response to Epstein-Barr virus,” said Yolken. “This indicated that the prevention and treatment of Epstein-Barr virus might represent an approach for the prevention and treatment of serious psychiatric disorders such as schizophrenia.”

Currently, there are no treatments available for EBV approved by the Food and Drug Administration, but a number of compounds that may prevent or treat replication of the virus are under investigation. The researchers considered the development of these approaches a high priority so that people with schizophrenia or other disorders associated with susceptibility to EBV could use them. In the meantime, researchers recommend preventing EBV transmission through good hygienic practices such as hand-washing and avoiding oral contact, such as kissing, with infected people.

https://www.hopkinsmedicine.org/news/newsroom/news-releases/2019/01/schizophrenia-linked-with-abnormal-immune-response-to-epstein-barr-virus

Teratogenesis, perinatal, and neurodevelopmental outcomes after in utero exposure to antiseizure medication

Pack AM, Oskoui M, et al. Teratogenesis, Perinatal, and Neurodevelopmental Outcomes After In Utero Exposure to Antiseizure Medication. Epilepsy Currents. 2025;0(0). doi:10.1177/15357597241258514
Abstract

This practice guideline provides updated evidence-based conclusions and recommendations regarding the effects of antiseizure medications (ASMs) and folic acid supplementation on the prevalence of major congenital malformations (MCMs), adverse perinatal outcomes, and neuro-developmental outcomes in children born to people with epilepsy of childbearing potential (PWECP). A multidisciplinary panel conducted a systematic review and developed practice recommendations following the process outlined in the 2017 edition of the American Academy of Neurology Clinical Practice Guideline Process Manual. The systematic review includes studies through August 2022. Recommendations are supported by structured rationales that integrate evidence from the systematic review, related evidence, principles of care, and inferences from evidence. The following are some of the major recommendations. When treating PWECP, clinicians should recommend ASMs and doses that optimize both seizure control and fetal outcomes should pregnancy occur, at the earliest possible opportunity preconceptionally. Clinicians must minimize the occurrence of convulsive seizures in PWECP during pregnancy to minimize potential risks to the birth parent and to the fetus. Once a PWECP is already pregnant, clinicians should exercise caution in attempting to remove or replace an ASM that is effective in controlling generalized tonic-clonic or focal-to-bilateral tonic-clonic seizures. Clinicians must consider using lamotrigine, levetiracetam, or oxcarbazepine in PWECP when appropriate based on the patient’s epilepsy syndrome, likelihood of achieving seizure control, and comorbidities, to minimize the risk of MCMs. Clinicians must avoid the use of valproic acid in PWECP to minimize the risk of MCMs or neural tube defects (NTDs), if clinically feasible. Clinicians should avoid the use of valproic acid or topiramate in PWECP to minimize the risk of offspring being born small for gestational age, if clinically feasible. To reduce the risk of poor neurodevelopmental outcomes, including autism spectrum disorder and lower IQ, in children born to PWECP, clinicians must avoid the use of valproic acid in PWECP, if clinically feasible. Clinicians should prescribe at least 0.4 mg of folic acid supplementation daily preconceptionally and during pregnancy to any PWECP treated with an ASM to decrease the risk of NTDs and possibly improve neurodevelopmental outcomes in the offspring.

Karakis I. “In the Name of the Father: Risk to the Offspring From Paternal Exposure to Valproate During Conception”. Epilepsy Currents. 2025;0(0). doi:10.1177/15357597251317286

Importance: Concerns exist about teratogenic and long-term neurodevelopmental outcomes of paternal use of valproate during spermatogenesis. Objective: To evaluate the association between paternal use of valproate during spermatogenesis and offspring risk of congenital malformations and neurodevelopmental disorders. Design, setting, and participants: This nationwide cohort study included 1,235,353 singletons born in Denmark between January 1, 1997 and December 31, 2017, identified in the Medical Birth Register; 1336 children had fathers who had filled prescriptions for valproate during spermatogenesis. Congenital malformations were identified in the first year of life and neurodevelopmental disorders were identified from 1 year of age until December 31, 2018. Statistical analysis was performed March 2024. Exposures: Paternal valproate exposure was defined as fathers who filled 1 or more prescriptions for valproate immediately before or during the time of spermatogenesis (ie, 3 months prior to conception). Main outcomes and measures: Children with major congenital malformations in the first year of life and with neurodevelopmental disorders before death or end of follow up were identified in Danish health registers. Log-binomial regression was used to estimate adjusted relative risks (ARRs) of congenital malformations, and Cox proportional hazards regression was used to estimate adjusted hazards ratios (AHRs) of neurodevelopmental disorders, adjusted for relevant confounders. Results: Among 1,235,353 live births (634,415 boys [51.4%] and 600,938 girls [48.6%]), 1336 children (0.1%) had fathers who filled prescriptions for valproate during spermatogenesis. The median follow up was 10.1 years (interquartile range [IQR] 5.1-14.8 years) for valproate-exposed children and 10.3 years (IQR 5.2-15.6 years) for valproate-unexposed children. A total of 43,903 children (3.6%) received a diagnosis of major congenital malformations in the first year of life, and 51,633 children (4.2%) received a diagnosis of neurodevelopmental disorders during follow up. When comparing the risk among valproate-exposed children with that among unexposed children, the ARR of major congenital malformations was 0.89 (95% confidence interval [CI] 0.67-1.18), the AHR of neurodevelopmental disorders was 1.10 (95% CI 0.88-1.37), and the AHR of autism spectrum disorder was 0.92 (95% CI 0.65-1.30). In analyses addressing the robustness of the findings (ie, dose-response analyses, sibling analyses, analyses restricted to children of fathers with epilepsy, analyses that used children with paternal lamotrigine exposure as active comparator, and analyses that used children with paternal exposure to valproate only before spermatogenesis as a negative control exposure), there still was no increased risk of any of the included end points. Conclusions and relevance: In all analyses based on this large Danish cohort study, results suggest that exposure to valproate during spermatogenesis was not associated with offspring risk of congenital malformations or neurodevelopmental disorders, including autism spectrum disorder.

Lennox-Gastaut syndrome treatment and prevention

Warren AEL, Patel AD, Helen Cross J, et al. Mobilizing a New Era in Lennox-Gastaut Syndrome Treatment and Prevention. Epilepsy Currents. 2025;0(0). doi:10.1177/15357597251321926

Abstract

This review summarizes content presented at the Pediatric State of the Art Symposium held during the American Epilepsy Society's annual meeting in December 2024. The symposium focused on Lennox-Gastaut syndrome (LGS), a severe developmental and epileptic encephalopathy that emerges in childhood. Despite its diverse etiologies, LGS is defined by a convergent constellation of electroclinical features: multiple seizure types including tonic seizures, slow spike-wave and generalized paroxysmal fast activity on EEG, and intellectual disability. LGS is almost always refractory to available therapies and accounts for substantial costs—in healthcare spending and in quality of life for affected individuals, their families, and caregivers. The symposium highlighted recent breakthroughs in research, clinical care, and outcome measurement that have positioned the clinical, scientific, and patient advocacy communities to usher in a new, more hopeful era of treatment and prevention.

Excerpts

How might preclinical models inform our understanding of LGS pathogenesis and treatment? Promising examples include the recent discovery that increased myelin plasticity exacerbates epilepsy progression in an SCN8A Scn8a+/- mouse model of generalized epilepsy, and that this epilepsy progression can be prevented by suppressing activity-dependent myelination. In zebrafish, high-throughput drug screens in models of Dravet syndrome identified Clemizole, a modifier of serotonin signaling, with potent anti-seizure effects; this has led to ongoing human trials for Clemizole as an adjunctive treatment for Dravet syndrome and LGS (ClinicalTrials.gov IDs: NCT04462770, NCT05066217). Recent studies in which other genes, including some linked to LGS, are engineered into zebrafish have demonstrated their utility as epilepsy models...

Addressing the Under-Utilization of Surgery for LGS

Surgery can be a highly effective treatment for medically refractory epilepsy, but there is increasing recognition that it is underutilized and often delayed in LGS, particularly in those who carry etiologies traditionally perceived as surgically non-remediable (e.g., non-structural genetic causes). In published studies of LGS, the lag time between seizure onset and surgery can be 20 years or more, often after numerous ASM attempts. This contrasts with ILAE guidelines stating that referral for surgical evaluation should occur as soon as drug resistance is ascertained, defined as the failure of adequate trials of two tolerated and appropriately chosen ASMs.

Conclusions
LGS is regularly described as “catastrophic” or “devastating.” While these terms capture the real and profound challenges faced by individuals and caregivers, they risk overshadowing the substantial progress made in understanding and managing LGS. This symposium highlighted recent advances across eight key areas, including the conceptualization of LGS as a secondary network epilepsy, the innovative use of electronic medical data to perform natural history studies and identify gaps in treatment coverage, and the development of preclinical models to explore pathogenesis and treatment. It also reviewed improvements in evidence-based treatment strategies, spanning pharmacotherapies, neuromodulation, surgery, and preventive approaches, alongside a growing emphasis on non-seizure outcomes and tailored assessment tools to measure meaningful, incremental progress. Together, these efforts mark a turning point toward more comprehensive and patient- and caregiver-centered care.

Botulinum toxin in the treatment of tics

Pandey S, Srivanitchapoom P, Kirubakaran R, Berman BD. Botulinum toxin for motor and phonic tics in Tourette's syndrome. Cochrane Database Syst Rev. 2018 Jan 5;1(1):CD012285. doi: 10.1002/14651858.CD012285.pub2. PMID: 29304272; PMCID: PMC6491277.

Abstract

Background: Gilles de la Tourette syndrome, or Tourette's syndrome, is defined as the presence of both motor and vocal (phonic) tics for more than 12 months, that manifest before the age of 18 years, in the absence of secondary causes. Treatment of motor and phonic tics is difficult and challenging.

Objectives: To determine the safety and effectiveness of botulinum toxin in treating motor and phonic tics in people with Tourette's syndrome, and to analyse the effect of botulinum toxin on premonitory urge and sensory tics.

Search methods: We searched the Cochrane Movement Disorders Group Trials Register, CENTRAL, MEDLINE, and two trials registers to 25 October 2017. We reviewed reference lists of relevant articles for additional trials.

Selection criteria: We considered all randomised, controlled, double-blind studies comparing botulinum toxin to placebo or other medications for the treatment of motor and phonic tics in Tourette's syndrome for this review. We sought both parallel group and cross-over studies of children or adults, at any dose, and for any duration.

Data collection and analysis: We followed standard Cochrane methods to select studies, assess risk of bias, extract and analyse data. All authors independently abstracted data onto standardized forms; disagreements were resolved by mutual discussion.

Main results: Only one randomised placebo-controlled, double-blind cross-over study met our selection criteria. In this study, 20 participants with motor tics were enrolled over a three-year recruitment period; 18 (14 of whom had a diagnosis of Tourette's syndrome) completed the study; in total, 21 focal motor tics were treated. Although we considered most bias domains to be at low risk of bias, the study recruited a small number of participants with relatively mild tics and provided limited data for our key outcomes. The effects of botulinum toxin injections on tic frequency, measured by videotape or rated subjectively, and on premonitory urge, are uncertain (very low-quality evidence). The quality of evidence for adverse events following botulinum toxin was very low. Nine people had muscle weakness following the injection, which could have led to unblinding of treatment group assignment. No data were available to evaluate whether botulinum injections led to immunoresistance to botulinum.

Authors' conclusions: We are uncertain about botulinum toxin effects in the treatment of focal motor and phonic tics in select cases, as we assessed the quality of the evidence as very low. Additional randomised controlled studies are needed to demonstrate the benefits and harms of botulinum toxin therapy for the treatment of motor and phonic tics in patients with Tourette's syndrome.

Moretti A. Is botulinum toxin effective and safe for motor and phonic tics in patients affected by Tourette syndrome? A Cochrane Review summary with commentary. Dev Med Child Neurol. 2020 Mar;62(3):274-276. doi: 10.1111/dmcn.14472. Epub 2020 Jan 20. PMID: 31957864.

Commentary on the above Cochrane review.

Kwak CH, Hanna PA, Jankovic J. Botulinum toxin in the treatment of tics. Arch Neurol. 2000 Aug;57(8):1190-3. doi: 10.1001/archneur.57.8.1190. PMID: 10927800.

Abstract

Objective: To evaluate the safety and efficacy of botulinum toxin A (BTX) injections in the treatment of tics in patients with Tourette syndrome (TS).

Background: BTX is an effective treatment for an increasing number of conditions characterized by abnormal muscle contractions. BTX may improve not only the motor component of tics, but also premonitory sensations that precede tics.

Methods: Thirty-five patients (30 male, 5 female) were treated with BTX in the sites of their most problematic tics. Response to BTX was based on a 0 to 4 clinical rating scale (0, no improvement, to 4, marked improvement in both severity and function). Questionnaires were administered to evaluate patients' impressions of overall efficacy and degree of benefit with premonitory sensations.

Results: Mean duration of tics prior to initial injection was 15.3 years (range, 1-62 years) and mean duration of follow-up was 21.2 months (range, 1. 5-84 months). The mean peak effect response in 35 patients treated in 115 sessions was 2.8 (range, 0-4); the mean duration of benefit was 14.4 weeks (maximum, 45 weeks); and the mean latency to onset of benefit was 3.8 days (maximum, 10 days). Twenty-one (84%) of 25 patients with premonitory sensations derived marked relief of these symptoms (mean benefit, 70.6%). Total mean dose was 502.1 U (range, 15-3550 U); mean number of visits, 3.3 (range, 1-16); and mean dose per visit, 119.9 U (range, 15-273 U). Sites of injections were as follows: cervical or upper thoracic area (17), upper face (14), lower face (7), vocal cords (4), upper back and/or shoulder (3), scalp (1), forearm (1), leg (1) and rectus abdominis (1). Complications included neck weakness (4), dysphagia (2), ptosis (2), nausea (1), hypophonia (1), fatigue (1), and generalized weakness (1), which were all mild and transient.

Conclusions: Botulinum toxin A injections are an effective and well-tolerated treatment of tics. In addition to improving the motor component of tics, BTX also provides relief of premonitory sensations.