The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne ...

which is absent in patients with Duchenne muscular dystrophy, and the protein Dysferlin, which is lacking in individuals with ...

Pronounced hyperaldolasemia and hypercreatine-phosphokinasemia have been shown to occur in the Duchenne type of muscular dystrophy before any detectable signs of muscle weakness, 3–6 and thus ...

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

According to the Mayo Clinic, muscular dystrophy is a group of diseases that cause loss of muscle mass and progressive ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

A new technology that delivers two halves of a gene separately could enable gene therapy treatments of muscular dystrophies.

The US biopharma will discontinue development of the Exondys 51 (eteplirsen) follow up treatment to focus on other treatment ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

Sarepta Therapeutics is advancing SRP-9003 gene therapy for LGMD, with phase 3 study results expected in early 2025. Learn ...

A small trial of an experimental vaccine has shown promising results for patients with the most aggressive type of breast ...