The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AOC 1044, an experimental therapy for…
Articles by Marisa Wexler, MS
EDG-5506, an oral treatment being developed by Edgewise Therapeutics, has been granted fast track designation for Duchenne…
Cognitive function in boys with Becker muscular dystrophy (BMD) appear to be stable over time, although these children may…
SRP-5051 is able to increase the production of dystrophin protein in people with Duchenne muscular dystrophy (DMD) amenable…
SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) being developed by Solid Biosciences, has been granted…
New trial data announced by Dyne Therapeutics show two experimental treatments for muscular dystrophy from the company’s FORCE…
People with Becker muscular dystrophy (BMD) caused by specific mutations tend to be younger when they first start using…
The European Commission has approved Agamree (vamorolone), a dissociative corticosteroid — a treatment expected to have fewer side effects…
The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease designations to…
Satellos Bioscience is hoping to bring SAT-3247, its newly nominated lead treatment candidate for Duchenne muscular dystrophy (DMD),…