ENDEAVOR, gene therapy for DMD
Sarepta Therapeutics’ investigational gene therapy for the treatment of Duchenne muscular dystrophy, SRP-9001, demonstrates robust expression and consistent safety profile using Sarepta’s commercial process material. The first 11 participants […]
Positive clinical results from phase 2 MOMENTUM study
Sarepta Therapeutics reports positive clinical results from phase 2 MOMENTUM study of SRP-5051 in patients with Duchenne muscular dystrophy amenable to skipping exon 51 Results suggest a highly potent next-generation […]
Genethon announces First Patient dosed in Clinical Trial
Genethon announces First Patient dosed in Clinical Trial of Investigational Gene therapy GNT004 for Duchenne Muscular Dystrophy. Communiqué de presse original > Genethon A first participant was dosed at I-Motion, […]
Pamrevlumab for the treatment of DMD
FibroGen receives Fast Track designation from the U.S. FDA for Pamrevlumab to treat Duchenne muscular dystrophy (DMD). April 12, 2021, Original press release. Source: FibroGen, Inc FibroGen, Inc. announced […]
Solid Biosciences reports efficacy and safety data
Solid Biosciences reports efficacy and safety data from the ongoing IGNITE DMD clinical trial and resumption of patient dosing in the 2E14 VG/kg cohort. La Force is sharing this press […]
Article about DMD in Nature.com
Recent studies on Duchenne muscular dystrophy (DMD) have greatly deepened our understanding of the primary and secondary pathogenetic mechanisms. Guidelines for the multidisciplinary care for DMD that address obtaining a […]
