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

The mother of a teenage boy, with muscular dystrophy, said her son hasn’t been able to leave their Oakland home for almost a ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

Kyle Albertson, an FFA alumnus and Purdue graduate, shares how FFA prepared him for a career in business despite his ...

One of the major highlights of the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is that invaluable networking opportunities can help clinicians elevate their own best ...

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

Researchers focused on Myotonic Dystrophy 1 heart problems are testing a novel approach to restore normal function.

The biotech stock's shares surged after Barron's said the market is underestimating potential sales of its Duchenne Muscular ...

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

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

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