The first person with limb-girdle muscular dystrophy type 2I/R9 (LGMD 2I/R9) has been dosed with the gene therapy AB-1003…
Articles by Marisa Wexler, MS
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…
An advisory committee of the U.S. Food and Drug Administration (FDA) has voted narrowly in favor of accelerated approval for…
An advisory committee of the U.S. Food and Drug Administration (FDA) will be meeting Friday to discuss SRP-9001 (delandistrogene…
After two years of treatment with losmapimod, people in a clinical trial with facioscapulohumeral muscular dystrophy (FSHD) showed…
Motor function improvements seen in boys with Duchenne muscular dystrophy (DMD)…