While a Phase 3 clinical trial of Elevidys (delandistrogene moxeparvovec-rokl) has failed to meet its main goal, the results…
Articles by Marisa Wexler, MS
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…
The gene therapy RGX-202 at lower dose was well tolerated by three boys with Duchenne muscular dystrophy…
Capricor Therapeutics intends to ask the U.S. Food and Drug Administration (FDA) to approve its experimental cell therapy…
A Phase 2 clinical trial testing the investigational oral medication EDG-5506 in people with Becker muscular dystrophy (BMD) is…
The conditional approval of Translarna (ataluren) in Europe as a treatment for Duchenne muscular dystrophy (DMD) — in…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy…