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 […]
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 of Investigational Gene therapy GNT004 for Duchenne Muscular Dystrophy. Communiqué de presse original > Genethon A first participant was dosed at I-Motion, […]
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 from the ongoing IGNITE DMD clinical trial and resumption of patient dosing in the 2E14 VG/kg cohort. La Force is sharing this press […]
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 […]
Santhera Announces Completion of First 6-Month Period of Pivotal VISION-DMD Trial with Vamorolone in Duchenne Muscular Dystrophy Original Press release > Pratteln, Switzerland, March 3, 2021 Santhera Pharmaceuticals announces that […]
Sarepta Therapeutics announces FDA approval of AMONDYS 45 (casimersen) injection to treat Duchenne muscular dystrophy (DMD) in patients amenable to skipping exon 45. Feb. 25, 2021, >Original press release< Sarepta […]
Italfarmaco provides an update on Givinostat, an ongoing clinical program, in an oral presentation at XVIII International Conference on Duchenne and Becker Muscular Dystrophy. Feb. 22, 2021, > Original press release […]
We wish to emphasize that the information contained in this article comes mainly from the RQMO website, the CORD website and the Rare Disease Day website. We thank them. February 28th is Rare disease day 2021. […]
