With recent improvements in healthcare, individuals with Duchenne muscular dystrophy (DMD) have prolonged life expectancy, and it is therefore vital to preserve their independence. Hand function plays ...

The company said it was “surprised” by the decision, which followed the ouster of cell and gene therapy officials from the ...

The FDA rejected a Duchenne muscular dystrophy cell therapy from Capricor Therapeutics, as a larger study of the treatment ...

Capricor Therapeutics said on Friday the U.S. Food and Drug Administration has declined to approve its cell therapy deramiocel for a heart condition associated with Duchenne muscular dystrophy.

In an opinion article, investigators argue that gene therapies may not achieve their full potential if the innate inflammation in Duchenne muscular dystrophy is not effectively managed.

A second Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), Sarepta Therapeutics said Sunday.

Dysfunctional dystrophin, the muscle protein affected in Duchenne, leads to an important choice for patients about how to ...

Edgewise Therapeutics, Inc., a leading muscle disease biopharmaceutical company, unveiled positive results in its sevasemten programme for Becker and Duchenne muscular dystrophies. The company ...

Genetics Duchenne muscular dystrophy (DMD) is a genetic disorder caused by mutations in the DMD gene—located on the X chromosome and representing the largest known human gene with 2.4 million base ...

Sarepta Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy treatments before two patients died, a proposed class action said. The ...

The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for sevasemten in Becker muscular dystrophy.

About Duchenne Duchenne, a severe degenerative muscle disorder, is the most common type of muscular dystrophy with a median life expectancy of around 30 years. Genetic mutations in the dystrophin gene ...