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 […]
Sarepta today announced topline results from Part 2 of Study 102, an ongoing, randomized, double-blind, placebo-controlled clinical trial evaluating its investigational gene transfer therapy SRP-9001 in Duchenne patients. In […]
A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From SRP-9001 in Participants With Duchenne Muscular Dystrophy Sarepta is now expanding their trial of SRP-9001 (ENDEAVOR) to […]
The 22nd International Annual Congress of the World Muscle Society took place in St Malo, between October 3 – 7, 2017. There were multiple breaking news stories on Duchenne muscular […]
Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) […]
My husband Paul and I have dreamed of this day since our son Hawken was diagnosed with Duchenne almost 15 years ago! We are overwhelmed with joy to announce […]
We are sharing the following update from Sarepta Therapeutics. Sarepta Therapeutics is updating the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study […]
