We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new […]
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This webinar discusses a groundbreaking gene therapy treatment for Duchenne Muscular Dystrophy (DMD), focusing on a method to introduce a miniaturized version of the dystrophin gene using a viral vector. […]
Here’s What You Missed at the MDA Clinical and Scientific Conference 2024
The FDA will make a decision whether or not to grant full approval of Sarepta’s gene therapy, Elevidys, by June 21,2024.
Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51. In part B of the Phase 2 MOMENTURM study, ambulatory and […]
While there is currently no cure for Duchenne muscular dystrophy, gene therapy has the potential to target the underlying genetic cause of many mono genetic diseases and could potentially benefit […]
My name is Debra Miller, founder and CEO of CureDuchenne, and the mother of an incredible son, Hawken, who has Duchenne muscular dystrophy. I’m here today to represent the voice of patient advocacy organizations serving those impacted by Duchenne – all of whom are in support of the accelerated approval of SRP-9001.
Sarepta Therapeutics is currently running multiple clinical trials in a few different countries. They are at various stages and for a range of ages. Sarepta issued an update for the […]