Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Duchenne muscular dystrophy (DMD) is the most common type. It’s caused by flaws in the gene that controls how the body keeps muscles healthy. The disease almost always affects boys, and symptoms ...

Characterized by progressive muscle degeneration, this X-linked disorder results from mutations in the DMD gene, which encodes dystrophin, a protein essential for maintaining muscle cell integrity.

Aug. 24, 2023 — Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored ...

INDIANAPOLIS (WISH) — It’s a devastating diagnosis that changes a child’s life forever, Duchenne muscular dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys.

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Updated ...

Delandistrogene moxeparvovec (Elevidys; Sarepta Therapeutics) appeared to protect muscle from progressive damage in patients ...

Novartis has reached an agreement to take control of Kate Therapeutics, a developer of gene therapies for inherited neuromuscular diseases, including Duchenne muscular dystrophy (DMD). The ...

PBGENE-DMD is the first in vivo gene editing program that has the potential to transform the treatment paradigm and deliver durable functional improvement for most patients, as up to 60% of those ...

The FDA approved Elevidys in June 2023 for ambulatory 4- and 5-year-old patients with a confirmed DMD gene mutation. A year later, the agency expanded the indication to include both ambulatory and ...