Treatment with PF-06939926, an experimental gene therapy for Duchenne muscular dystrophy (DMD), was generally well-tolerated among ambulatory patients —…
Articles by Marisa Wexler, MS
The investigational gene therapy SRP-9003 was well tolerated overall and improved measures of physical functioning for up to three…
The corticosteroid Emflaza outperforms prednisone at slowing disease progression in people with Duchenne muscular dystrophy (DMD), according to…
Treatment with BBP-418 improved walking ability among people with limb-girdle muscular dystrophy type 2i (LGMD2i) in a Phase 2…
A potentially pivotal Phase 3 clinical trial called HOPE-3, which will test the effectiveness of the experimental cell therapy…
A protein called GCN5 is vital for normal muscle health and helps to coordinate the expression of several important muscle…
Children with Duchenne muscular dystrophy (DMD) who had been given a placebo in a clinical trial experienced a marked…
Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for…
Atamyo Therapeutics has been given the go-ahead to start clinical testing of ATA-100, the company’s investigational gene therapy for a specific…
Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S.