Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

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

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

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...

4. Administered the first dose of a new gene therapy for muscular dystrophy. 5. Implemented an Omnicell inventory management program at WVU Medicine's Mary Babb Randolph Cancer Center, achieving ...

Soaking up the Aussie sun, the actor, who grew up in Wales, showcased his muscular torso as he went shirtless while wearing a pair of navy shorts. The star is best known for his breakout role ...

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.

Duchenne muscular dystrophy is a genetic disorder characterized by the progressive loss of muscle mass, due to mutations in the dystrophin gene. Without the corresponding functional protein ...

If you’re living with Duchenne muscular dystrophy (DMD), planning ahead can help support the success of future education, employment, and housing efforts. Duchenne muscular dystrophy (DMD ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is ...

Scientists report results from a promising new approach to treat the rare neurodegenerative disorder. Spinal muscular atrophy (SMA) is a progressive neurodegenerative disorder set in motion before ...

A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available in Northern Ireland. Alfie, from Newry, County Down, was diagnosed ...