Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...

When Ingram became Sarepta Therapeutics’ CEO in 2017, he didn’t have a connection to muscular dystrophy, but he has developed ...

More than 30 types of muscular dystrophy have been identified. They all cause problems with movement due to muscle protein defects caused by genetic mutations. Muscular dystrophy is a group of ...

KYOTO--Utilizing genome editing technology, a team of researchers has developed a much more effective treatment for a certain type of muscular dystrophy. The method has recently been completed by the ...

On World Rare Disease Day, we take a look at Pompe disease - a rare genetic disorder caused by a deficiency of the GAA enzyme, leading to glycogen buildup in muscles. It results in progressive muscle ...

Becker muscular dystrophy (BMD) is a type of muscular dystrophy, a genetic condition that weakens and damages muscles. It can worsen with age. However, its symptoms are less severe than those of ...

A multidisciplinary team, usually led by a neurologist, treats muscular dystrophy (MD). They’ll tailor the type of treatment you receive to your specific needs. MD is a group of conditions that lead ...

After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales of Duchenne muscular dystrophy gene therapy Elevidys continue to decline.

ST. LOUIS — On a beautiful day in Forest Park, dozens of people brought hope to kids and adults with Muscular Dystrophy by just taking a long walk. The Muscular Dystrophy Association used the walk to ...

Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...

Sen. Roger Wicker urges states to add Duchenne muscular dystrophy to newborn screening, after federal health officials back ...