Dosing has begun in a Phase 2 clinical trial evaluating PepGen‘s PGN-EDO51 — a treatment candidate for Duchenne…
Articles by Steve Bryson, PhD
Deleting exon 51 in the DMD gene — which mimics exon 51 skipping therapies — reversed all signs of…
Various factors influenced the results of the creatine kinase-MM (CK-MM) blood test used to screen infants for Duchenne muscular…
In people with Becker muscular dystrophy (BMD), both an elevated creatine-to-creatinine ratio and lower myostatin muscle protein levels in…
Levels of titin, a muscle protein, were found to be elevated in the urine of boys with Duchenne muscular…
Microdystrophin gene therapy effectively maintained long-term heart function in a mouse model of severe Duchenne muscular dystrophy (DMD), a…
DYNE-251, an investigational therapy for Duchenne muscular dystrophy (DMD), has received orphan drug and rare pediatric disease designations from…
The approved antiparasitic medicine pentamidine, loaded into biocompatible and biodegradable nanoparticles, successfully eased signs of myotonic dystrophy type 1…
Treatment with AOC 1001 eased myotonia, when muscles are unable to relax after a contraction, in adults with myotonic…
Vamorolone, an investigational therapy for Duchenne muscular dystrophy (DMD), maintained biomarkers of bone formation and turnover compared to…