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 ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying therapies using gene replacement, antisense oligonucleotides, and gene transfer ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

A new mouse model mimicking the liver symptoms of myotonic dystrophy type 1 -- the most prevalent form of adult-onset muscular dystrophy -- provides insight into why patients develop fatty liver ...

Physical activity and rehabilitation are key components of Duchenne muscular dystrophy treatment, despite recent drug therapy advances. "Rehabilitation care and rehabilitation teams with experience ...

After a rocky 2025, Sarepta Therapeutics’ executives admit they have work to do to bring patients back into the fold as sales ...

Facioscapulohumeral muscular dystrophy (FSHD) is a genetic condition that can lead to the slow deterioration of muscles, predominantly in the face, shoulder blades, and upper arms. Facioscapulohumeral ...

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

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 ...

Del-desiran reduced DMPK mRNA levels, but led to two serious adverse events ...