RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

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

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

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

Biopsy data from eight of the boys showed a 6.4% mean muscle content-adjusted dystrophin ... benefit seen one year after taking Sarepta’s DMD gene therapy Elevidys. The 48-week data for Wave ...

Avidity Biosciences, Inc.'s innovative AOC platform and promising clinical programs make it a high-risk, high-reward ...

The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...

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

PBGENE-DMD-edited dystrophin mRNA transcript was detected in PAX7+ cells, a marker for muscle satellite stem cells, suggesting potential for durable therapeutic effects compared to standard gene ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin ... to produce the protein known as dystrophin, it is this protein that ...