Full article available through PubMed and Wiley.
https://onlinelibrary.wiley.com/doi/10.1002/jimd.12305
10 CONCLUSION
I started this wave of support for MSD research hoping that Dylan could benefit in his lifetime from our efforts, and this hope remains. I also knew that many MSD patients and their families across the world could benefit if we can ensure science delivers a treatment. I will endeavor to do my best to assist the other MSD foundations that might wish to lean on my experience. Especially with regard to identifying research gaps and funding research in the future. I expect that together we can make life-changing differences for patients. I anticipate that funding from the various MSD charities can complement research initiatives that are underway and that show the most potential.
I wish that one day there will be treatment options available to MSD patients and that their families can appreciate it and enjoy a better life with their children. Without knowing the struggle and huge sacrifices by many, that it took to get treatments is something I wish my family could have had! There are many difficulties with trying to develop potential therapies for ultra-rare diseases that I realized in time due to my journey with MSD. There could be an element of bliss in not knowing these difficulties. If I ever get a call, one day, from a parent of a child affected by MSD to thank me for my efforts when there is a treatment… I would simply say that it was the many people who believed and supported our cause are the ones that really made it happen.
Family July 2019
I have invested so much of myself in MSD research I cannot ever see myself being able to walk away. I want to witness the first child being treated, with the efforts that have gone in this would be a very emotional experience! Many research initiatives have moved on and real progress has been made and for this I am so grateful. However, when you have a child that is affected by something as relentless as MSD, research is never fast enough. You have no option but to witness a decline in your child, which is soul destroying, while you try to do your best for them due to love. Having a child affected by MSD, and no treatment, is like starting with a piece of rock, every day a little bit gets chipped away, you cannot necessarily see it, but one day it will be gone.
10 CONCLUSION
I started this wave of support for MSD research hoping that Dylan could benefit in his lifetime from our efforts, and this hope remains. I also knew that many MSD patients and their families across the world could benefit if we can ensure science delivers a treatment. I will endeavor to do my best to assist the other MSD foundations that might wish to lean on my experience. Especially with regard to identifying research gaps and funding research in the future. I expect that together we can make life-changing differences for patients. I anticipate that funding from the various MSD charities can complement research initiatives that are underway and that show the most potential.
I wish that one day there will be treatment options available to MSD patients and that their families can appreciate it and enjoy a better life with their children. Without knowing the struggle and huge sacrifices by many, that it took to get treatments is something I wish my family could have had! There are many difficulties with trying to develop potential therapies for ultra-rare diseases that I realized in time due to my journey with MSD. There could be an element of bliss in not knowing these difficulties. If I ever get a call, one day, from a parent of a child affected by MSD to thank me for my efforts when there is a treatment… I would simply say that it was the many people who believed and supported our cause are the ones that really made it happen.
Family July 2019
I have invested so much of myself in MSD research I cannot ever see myself being able to walk away. I want to witness the first child being treated, with the efforts that have gone in this would be a very emotional experience! Many research initiatives have moved on and real progress has been made and for this I am so grateful. However, when you have a child that is affected by something as relentless as MSD, research is never fast enough. You have no option but to witness a decline in your child, which is soul destroying, while you try to do your best for them due to love. Having a child affected by MSD, and no treatment, is like starting with a piece of rock, every day a little bit gets chipped away, you cannot necessarily see it, but one day it will be gone.
No comments:
Post a Comment