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

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...

William is a seven-year-old boy from Southern Oregon living with Duchenne muscular dystrophy. Now, he is preparing to undergo a surgery that has never been tried before, a procedure designed ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD).

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

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

Share on Facebook. Opens in a new tab or window Share on Bluesky. Opens in a new tab or window Share on X. Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Eight therapies ...

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 ...

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

On Midday Minute for Wednesday Sen. John Carley has been found, a South Dakotan received the Medal of Honor and a local family is looking to find a cure for ...