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

There are some approved gene therapies for DMD, including Roche and Sarepta’s Elevidys (delandistrogene moxeparvovec-rokl), which gained accelerated approval by the US Food and Drug ...

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 ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

A new gene therapy for Duchenne muscular dystrophy (DMD) has shown promise in not only slowing the progression of the disease but potentially even reversing the muscle damage, with human trials ...

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 ...

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...

In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne muscular dystrophy. In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne ...

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 ...