A Phase 2 clinical trial testing the investigational oral medication EDG-5506 in people with Becker muscular dystrophy (BMD) is…
Articles by Marisa Wexler, MS
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…
Synthetic biology company bit.bio has launched two new human muscle cell models to advance research into new treatment strategies…
After a year of treatment with the experimental oral therapy EDG-5506 in a Phase 1 study, most men with…
Catalyst Pharmaceuticals has entered a licensing agreement for the North American rights to vamorolone, a dissociative corticosteroid under review…
Three advocacy organizations are teaming up to run a clinical trial to test if efgartigimod, an approved treatment for certain…
Avidity Biosciences is planning to double the dosage of AOC 1001 being given to a dozen people with…