A gene editing approach can restore dystrophin production in cell and animal models of Duchenne muscular dystrophy (DMD), according…
Articles by Marisa Wexler, MS
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…
A new project hopes to improve data collection related to Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy…
The first person with limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9) has been dosed with the gene therapy AB-1003…