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 […]
We’re pleased to share this letter and news from our partner Santhera about their plans for 2019! For more than 10 years, Santhera has been working on treatments for DMD, […]
Highlights from day three at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly. Mallinckrodt Pharmaceuticals Mallinckrodt provided an update on their Phase 2 […]
Highlights from day two at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly. Sarepta’s Gene Therapy Program: Sarepta provided a much-anticipated update on […]
Highlights from the first day at the World Muscle Society meeting in Mendoza, Argentina, provided by CureDuchnne’s scientist, Dr. Mike Kelly. Carrier-mothers: It’s recognized that Duchenne and Becker muscular dystrophy […]
Dear Friends, We are pleased to provide you with detailed information from Santhera Pharmaceuticals about an ongoing clinical trial that your son may be able to participate in. Beyond investing […]
PTC Therapeutics Update on Ataluren Study 041 PTC Therapeutics is enrolling boys and young men with a nonsense mutation in a clinical trial for Ataluren. To ensure that participants who […]
The third day of the conference focused on stem cells, new small molecule screens, poster sessions and clinical trial updates. Hirofumi Komaki (National Center of Neurology and Psychiatry, Japan) presented […]
The second day of the conference focused on gene editing, gene therapy as well as the latest research into the development of disease biomarkers. Presentations included: Dongsheng Duan (University of […]
