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

An Indiana man is proving that determination can break down barriers by starting his own company despite life's challenges.

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

At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X ...

Posters presented at the 2025 Muscular Dystrophy Association Clinical & Scientific Conference highlight the complex care ...

Children with SMA given the gene therapy Zolgensma in infancy are maintaining motor milestones after up to 10 years, new ...

Sarepta Therapeutics (SRPT) stock is reeling this week following the death of a 16-year-old patient treated with its flagship gene therapy, ...

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...

A grandmother joined her son and some family friends to abseil 100 feet down from the Falkirk Wheel to raise vital cash for ...