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. […]

Decoding Gene Therapy for Duchenne

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 […]

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Debra Miller Representing Patient Advocacy Community

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.  

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Sarepta Clinical Trials Community Bulletin

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 […]