An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...

In the last year, some drugmakers have pulled back from the sector, including Pfizer, which recently stopped selling its gene ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

While plenty of people are going green in honor of Saint Patrick's Day, one student at East Stroudsburg University is turning ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease-- --Data Presented at the ...