Monday, October 7, 2024

Denial of coverage for Zolgensma in twins with spinal muscular atrophy 1

Newborn twins with a rare — but often fatal — genetic disorder have been denied life-saving treatment by their family’s insurance company. 

Shortly after welcoming Eli and Easton Reed on March 31, 2024, the family tells KMBC that the twin boys were diagnosed with spinal muscular atrophy.

The genetic disorder can “damage and kill specialized nerve cells in the brain and spinal cord,” the National Institute for Neurological Disorders and Stroke explains.

While there is no cure, treatment includes “managing the symptoms and preventing complications.”  

And for the St. Joseph, Missouri, twins, treatment included the medication Zolgensma — which the family says their insurance company stopped covering just one day before the twins were born.

“Time is of the essence with this ... since they don’t have symptoms as of right now,” the twins’ mother, Amanda Reed, told KMBC. 


“It's best for them to receive this treatment now. because once symptoms start, it's un-reversible. So, time is of the essence, but we are still trying to explore all of our options.“

Out-of-pocket costs for the medication are “between $1 million and $2.5 million per child, a daunting amount that we are striving to secure through insurance appeals and your continued support,” a GoFundMe established to help them pay for the medication explains.

Zolgesnsma is a one-time treatment that “targets the genetic root cause of spinal muscular atrophy,” the website says, "stopping the progression" of the disease.

As the GoFundMe shared, the family’s insurance company “called an emergency meeting to review all of the information to decide whether or not they will allow their insurance to cover the gene therapy the boys need.”

But on April 26, the board denied their appeal, the GoFundMe says, leaving the family to pay for the life-saving therapy themselves.

“I’m holding my heart in my hands,” the twins’ father, Austin Reed, told KMBC.

“The fact that their life is in somebody’s else’s hands, whether they get this treatment or they don’t, you know, then that’s somebody else’s choice and that’s hard to cope with that right now.”

"Patients less than six months of age at disease onset will never sit independently and will likely to die of respiratory failure before two years of age," the National Library of Medicine says.

And as the Cleveland Clinic says, infants with type 1 SMA  — which means symptoms appear within the first 6 months of life — “usually die before their second birthday.”

https://people.com/insurance-denies-newborn-twins-fatal-disorder-treatment-8641768

Update 5/9/2024: Kecia Vant Hof, the organizer behind the GoFundMe page for Eli and Easton Reed, who were initially denied life-saving treatment for Spinal Muscular Atrophy (SMA), has announced that the twin newborns will be receiving the treatment next week.

In an update posted to the GoFundMe page, she wrote, “I want to personally thank each and every one of you for being so generous during my sister and her family’s time of need. Your love and encouragement has been such an incredible blessing.

“Due to all of you, the news media outlets who shared Eli and Easton’s story, and a powerful God, Eli and Easton WILL be receiving the gene therapy NEXT WEEK!! We are so blessed and thankful that the boys will have a fighting chance and we cannot thank you all enough.”

Vant Hof said that the GoFundMe has been closed but the $400,000 that was received so far will be used for any upcoming necessary treatments the boys may need.

Update 5/8/2024: Shortly after news broke that newborn twins Eli and Easton Reed had been denied the life-saving medication Zolgensma to combat Spinal Muscular Atrophy (SMA), the company that had denied the coverage, Mosaic Life Care, announced a new fund to help pay for the treatment. Mosaic Life Care, where the mother of Eli and Easton, Amanda Reed, works, is reported to have ended coverage of the life-saving drug that Eli and Easton need just one day after the boys were born and four days before they were diagnosed with SMA.

“Children’s Mercy, because that’s where we would be going for the treatment, tried to ask them if it could be covered since they were born a day before the cut, and they were denied,” Reed said. She added that the diagnosis and denial of coverage has “been a nightmare, an absolute nightmare.”

In a May 3 statement, Mosaic Life Care announced it established a fund to support treatment for rare genetic neuromuscular diseases such as SMA. It said it contributed $1.5 million to the fund after it received a $1.9 million anonymous donation. The money is said to be immediately available for the delivery of Zolgensma.

“We are pleased to announce the creation of a $3.4 million dollar philanthropic fund through the Mosaic Life Care Foundation for the purpose of supporting genetic treatment for rare genetic neuromuscular diseases such as Spinal Muscular Atrophy (SMA),” said Mosaic Life Care CEO Mike Poore. He added, “Using our extensive government, hospital and medical connections, we have been making exhaustive efforts behind the scenes to successfully secure substantial additional support for the treatment of SMA.”

5/3/2024: An insurance company is coming under fire for denying the coverage of life-saving treatment to a set of twins who will otherwise likely die before their second birthday.

People Magazine reported that Eli and Easton Reed were born on March 31, 2024, and shortly after they were both diagnosed via newborn screening with Spinal Muscular Atrophy (SMA), a progressive genetic disorder that kills nerve cells in the brain and spinal cord. The National Institute for Neurological Disorders and Stroke states that there are different forms, including Types I, II, III, and IV with Type I being the most severe. Without treatment, most children with Type I will die before the age of two as they progressively lose the ability to walk, swallow, and breathe.

However, new hope has come in the form of a single-dose treatment of a medication called Zolgensma, which is being called a “miracle drug.”

In 2021, Live Action News reported the story of baby Joseph Crew Delia who was diagnosed with SMA after birth and was given the one-time IV infusion of Zolgensma, which targets the genetic root cause of SMA and can prevent the deteriorating effects of the condition. The life-saving medication, however, costs $2.1 million. While Crew’s insurance covered $500,000 of it, his parents raised funds to cover the rest. Today, he’s a healthy toddler.

Eli and Easton Reed each need a dose, making the out-of-pocket price tag for their parents $4.2 million — and insurance is refusing to cover any of the cost.

“Time is of the essence with this … since they don’t have symptoms as of right now,” the twins’ mother, Amanda Reed, told KMBC. “It’s best for them to receive this treatment now. Because once symptoms start, it’s un-reversible. So, time is of the essence, but we are still trying to explore all of our options.”

A GoFundMe was created to help raise money for treatment while the boys’ parents appealed the insurance company’s decision. But on April 26, the insurance company denied the appeal.

“I’m holding my heart in my hands,” the twins’ father, Austin Reed, told KMBC.

“The fact that their life is in somebody else’s hands, whether they get this treatment or they don’t, you know, then that’s somebody else’s choice and that’s hard to cope with that right now.”

Without the treatment, the deteriorating effects of SMA could set in within six months, causing the boys to never be able to sit independently and likely die of respiratory failure by age two. SMA is progressive and Zolgensma can’t undo damage that is already done.

It stops the disease’s effects from progressing, so the sooner it is administered, the better.

https://www.liveaction.org/news/insurance-company-refuses-fund-newborns-care/

Two California parents are hoping to raise funds for the "most expensive drug in the world" to save the life of their daughter, who was recently diagnosed with a rare degenerative disease.

For parents Ceri and Rory Devine, things seemed to be going smoothly in the first months following the birth of their daughter, Rhys, in December 2019. But the couple noticed things were amiss when their baby girl noticeably fell behind in her development when she was 4 months old.

'That mother’s intuition was nagging me," Ceri, from Los Angeles, said on a website set up by the family. "She always struggled with tummy time, and wasn’t able to roll over or hold her head up for very long."

The couple took their daughter in for a check-up, and their pediatrician recommended physical therapy after examining Rhys' low muscle tone. But when she was reevaluated two months later, their doctor suspected Rhys had Spinal Muscular Atrophy. This rare genetic disease causes muscles to weaken throughout the body due to cellular defects in the brain stem and spinal cord.

What followed was "the longest two weeks" of the couple's lives, and after visiting numerous specialists, Rhys was officially diagnosed with SMA Type 1.

According to Boston Children's Hospital, SMA Type 1 (also known as Werdnig-Hoffmann disease) causes various symptoms in babies as their muscles waste away, such as being unable to sit without support and difficulty breathing, feeding and swallowing. There is no cure for the disease, and, tragically, many babies with SMA Type 1 die before the age of 2.

Despite the tragic news, Ceri and Rory were given some hope — doctors said there were two gene therapy treatments that could potentially improve Rhys' quality of life. But they wouldn't be easy to get.

One of the treatments, Spinraza, has shown improvement in children with SMA Type 1 and 2, according to SMA News Today. The drug — which is administered every four months through a spinal injection — costs $750,000 the first year and $375,000 every year after that for life, the parents say.

The other treatment, Zolgensma, was approved by the FDA in 2019 and is known as the "most expensive drug ever," NPR reported. It costs $2.1 million for a single dose, but the drug has shown improvement in young patients with SMA and "some patients are even able to walk independently," according to Ceri and Rory.

"Because SMA Type 1 is aggressive and degenerative, this is a race against time," the parents wrote on the website, Rise Up for Rhys.

"With each day that passes, motor neurons in Rhys’ sweet little body are becoming inactive," they continued. "In addition to the gene therapy, Rhys will need ongoing physical therapy and support equipment based on her needs to help her develop, improve mobility, and improve her quality of life."

The family started a GoFundMe campaign to raise funds for the therapies, and have raised over $273,000 as of Friday afternoon.

"Every child should have the best possible chance at life," the couple wrote. "Please join us to surround our happy little girl in love and support."

https://people.com/health/six-month-old-girl-with-rare-disease-in-need-of-life-saving-medicine-that-costs-2-1m/

See: https://www.facebook.com/riseupforrhys/

Rhys was treated with Zolgensma.





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