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

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

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 ...

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 ...

The study uncovers the molecular 'rules' driving the arrangement of emerin into nanoclusters and the mechanisms leading to their defective assembly in people with muscular dystrophy. A new ...

“Our technology has safely treated children as young as 18 months with conditions ranging from congenital muscular dystrophy to cerebral palsy, epilepsy, and autoimmune disorders,” said Donna ...