The U.S. Food and Drug Administration (FDA) granted accelerated approval to the gene therapy SRP-9001 (delandistrogene moxeparvovec) for children ages…
Articles by Marta Figueiredo, PhD
Boys with Duchenne muscular dystrophy (DMD) who cannot walk and have advanced scoliosis are at a higher risk of…
The FSHD Society has joined forces with the Critical Path Institute (C-Path) to facilitate the collection of data…
DT-DEC01, Dystrogen Therapeutics’ experimental chimeric cell therapy for Duchenne muscular dystrophy (DMD), appears generally safe and improved muscle…
An ion channel called Piezo1 regulates muscle stem cell (MuSC) states and the number of cells that respond quickly to…
Santhera Pharmaceuticals has entered into an agreement with SEAL Therapeutics, a spin-off of the Biozentrum of the University…
Significantly higher levels of sphingolipids — a type of fatty molecule involved in inflammation, scarring, and cell death — are…
Cognitive problems seen in some Duchenne muscular dystrophy (DMD) patients may be associated with a shift toward the amyloidogenic…
The U.S. Food and Drug Administration (FDA) has approved Regenxbio’s request to launch a Phase 1/2 clinical trial in…
The experimental oral therapy EDG-5506 is generally safe and results in rapid and profound reductions in the levels of muscle…