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 ...

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, ...

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

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

“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 ...

The muscular dystrophies are commonly associated with cardiovascular complications, including cardiomyopathy and cardiac arrhythmias. These complications are caused by intrinsic defects in ...

Spinal bulbar muscular atrophy (SBMA) causes a loss of motor neurons in the spinal cord and brainstem. It mainly affects facial and swallowing muscles and the muscles in the arms and legs.