People with Becker muscular dystrophy (BMD) caused by specific mutations tend to be younger when they first start using…
Articles by Marisa Wexler, MS
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),…
The expression of a protein called ACTC1 in muscle fibers is critical for muscle repair — and vital to the…
While a Phase 3 clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) has failed to meet its main goal, the results…
DYNE-251, an experimental treatment for Duchenne muscular dystrophy (DMD), is being well tolerated by patients in an ongoing clinical…
A gene editing approach can restore dystrophin production in cell and animal models of Duchenne muscular dystrophy (DMD), according…
A European Medicines Agency (EMA) committee has recommended that vamorolone be approved in the European Union to treat…
People with myotonic dystrophy type 1 (DM1) reported gains in measures of muscle strength after being treated with AOC 1001…