Researchers have discovered a new, unrecognized type of muscular dystrophy that’s caused by inherited mutations in the SNUPN gene,…
Articles by Steve Bryson, PhD
Up to eight years of Exondys 51 treatment (eteplirsen or AVI-4658) extended survival in Duchenne muscular dystrophy (Â鶹Éç)…
Regulators in France and Italy have given Atamyo Therapeutics the green light to launch a clinical trial testing ATA-200,…
A cell-based study revealed the biological mechanism behind the increased tendency for people with myotonic dystrophy type 2 (DM2)…
Dosing has begun in a Phase 2 clinical trial evaluating PepGen‘s PGN-EDO51 — a treatment candidate for Duchenne…
Deleting exon 51 in the Â鶹Éç 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 (Â鶹Éç), a…