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

Pediatric neurologist Omer Abdul Hamid is one of the few doctors in the US who administer gene therapy for Duchenne muscular ...

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.

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 ...

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 ...

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 ...

Multidisciplinary coordination is crucial for effective gene therapy administration in Duchenne muscular dystrophy, focusing on institutional readiness and patient-centered care. Guidelines ...

Following report of a patient’s death after treatment with Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys, some families have paused the treatment process to seek additional ...

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 ...