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 ...

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

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 ...

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

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 ...

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

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 ...