In myotonic dystrophy type 2 (DM2), muscle cells have dysfunctional mitochondria, the cell’s so-called powerhouse that’s key to energy…
Articles by Marisa Wexler, MS
Treatment with givinostat — an oral therapy currently up for approval in the U.S. and Europe — significantly slowed…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AOC 1044, an experimental therapy for…
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…