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.
CureDuchenne Ventures Invests in hC Bioscience and its Novel tRNA-Based Therapeutic Approach for Duchenne Muscular Dystrophy
We are pleased to announce that six sites are open for enrollment for the Canyon trial for individuals living with Becker Muscular Dystrophy! Canyon is a Phase 2 trial of […]
CureDuchenne, as an early investor in Avidity Biosciences, is excited to share the announcement of the Phase 1/2 EXPLORE44™ clinical trial of AOC 1044 in healthy volunteers and participants with […]
At the core of the MAA submission are positive data from the pivotal Phase 2b VISION-DMD study which comprised a (1) 24-week period to demonstrate efficacy and safety of vamorolone […]
On January 14th 2022, the U.S. FDA placed a clinical hold on Dyne’s Investigational New Drug (IND) submission for the launch of their clinical trial of DYNE-251, which targets Duchenne […]
CureDuchenne is happy to share the news that REGENXBIO will be launching a novel gene therapy trial in Duchenne during the first half of 2022. RGX-202 aims to deliver a […]
We are sharing a holiday message and community update from PTC Therapeutics. Dear Duchenne Community, As we enter the holiday season, I wanted to thank the community for all your […]
PTC Therapeutics held a community conference today regarding Ataluren. Stuart Peltz, President and CEO, PTC Therapeutics, Inc., welcomed and thanked the community as well as […]
