Wow. What a week our community had last week. By now, you’ve probably heard the momentous news that the FDA broadened the label for ELEVIDYS gene therapy to include older boys with Duchenne, ages 4 and up. As both the mom of an incredible man with Duchenne and an advocate for all boys and men with this disease that takes abilities away from our loved ones every day, this milestone resonates deeply with me. For families like ours, who have faced the relentless challenges of Duchenne, this moment is a testament to the progress we are making. All boys and young men, like my son Hawken, urgently need treatments to slow the progression of this devastating disease. We do not have the luxury of time to wait for a perfect solution. With limited or no options available for so many, any treatment that can help delay the disease's progression is a huge step forward. The ability to choose gene therapy provides a critical option for many families and their physicians to consider, offering a glimmer of hope where there was little before. I’m thankful to the companies, like Sarepta Therapeutics, that are driving treatments forward, and to the FDA for advancing these innovative treatments. And our community stands in eternal gratitude to the families who have bravely participated in clinical trials, knowing the risks and taking them on anyways on behalf of their child and thousands of others who stand to benefit. But we’re not done yet. There are limitations to this treatment that we must address, and individuals who will still be excluded from treatment. We are doubling down on our efforts with renewed confidence and determination. We have come so far, and with the wind at our backs, we know we will cure this disease. CureDuchenne
Well said Debra Miller. I share your gratitude and deep appreciation for the families that participate in clinical trials. These courageous individuals help us bring the science from the lab to the real world. These encouraging developments motivate us to push the science forward and close more treatment gaps.
Jeff Lansman
2w
Three FDA review teams and two top officials recommended Sarepta's application be rejected due to insufficient and conflicting clinical data. They were overruled by Marks, head of the FDA center that reviews gene therapies, who found the results supportive enough to broaden Elevidys' label.
Russ Reiss
4w
Thank you, Debra and CD for supporting the entire DMD community in making these important wins toward a cure possible.
Awesome work done by both of you! Thank you so much for all What you are doing for patients. Deb you are an example for me. All the Best for you and your family :-)
As a father with a 2 and a half year old with Duchenne, thank you for everything CureDuchenne and Sarepta has done to make these treatments possible.
Amazing work by these companies and the CureDuchenne team.
A step forward for permanent treatment....all the best!
Ginne Davis
2w
Great job, Debra!
Professor Emeritus of Cellular & Molecular Pharmacology, UCSF School of Medicine
2wElividys was rejected by three independent FDA Scientific Advisory Boards for 1) failure to meet significant clinical endpoints that changes the tragic trajectory of Duchennes; 2) failure to meet even minor surrogate endpoints of dystrophin minigene expression; 3) exposing children to an ineffective viral therapy which will trigger immune response to a possible future genetic effective therapy; 4) is based on a novel minigene which bears no resemblance to the milder Becker’s gene product. Political pressure led the FDA to override the independent Scientific Advisory Panels’ rejection, exposing children with Duchennes to a dangerous and ineffective gene therapy. Sarepta’s gene therapy is worthless unless you believe paying millions of dollars for a limited 2-3 second increase in walk time which can be a highly biased indicator with absolutely no change in disease trajectory or mortality. This is the worst of drug development but the MDA celebrates, FDA bigwigs keep their jobs, parents are given the appearance of hope and the affected children suffer the consequences of an ineffective treatment which makes it impossible for receiving a future drug that works: https://www.fda.gov/media/168021/download