The research team found that a specific muscle protein, known as MEF2C, is significantly reduced in muscle cells with CP.

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the ...

The treatment caused muscle cells to begin to divide and the mouse’s ... P400PM_186709); and Fondation Leducq. CITATION: “An Enhancer-Based Gene Therapy Strategy for Spatiotemporal Control of Cargoes ...

Opens in a new tab or window A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta ...

The therapy works by using a disabled virus to insert a gene that helps the body produce dystrophin, a protein crucial for muscle function. Sarepta said more than 800 patients have received ...

It’s the first gene therapy approved in the U.S. for the rare muscle-wasting condition, which causes weakness, loss of mobility and early death in males. The FDA granted full approval last year ...

When considering gene therapy, adults are “not just big kids,” she explained, because DMD decreases the muscle mass/body weight ratio over time, which correlates with age and ability to ...

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) has developed an innovative gene-therapy strategy ...

Elevidys, which costs $3.2 million for a one-time infusion, was the first gene therapy approved in the U.S. for Duchenne muscular dystrophy, a rare condition that causes muscle weakness ...