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

Delandistrogene moxeparvovec, a gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD), was found ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment.

Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

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

Researchers from the University of Queensland recently provided details on the discovery and preclinical characterization of ...

Preservation of Skeletal Muscle Function Shown Over 3 Years Resulting in 52% Slowing of Disease----Data Presented at the 2025 Muscular ...

And it's first such treatment in his class," said ... Karachunski said there are many more muscular dystrophy treatments that are currently in clinical trials that he believes could help Colton ...

SAN DIEGO, March 17, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare ...

The patient in this case had suffered acute liver failure following the Elevidys infusion. Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy (DMD) who received ...