Kuntz, MD, was a fellow about 45 years ago, there were few more devastating diagnoses than Duchenne muscular dystrophy (DMD).

Sarepta will discontinue its next generation of exon-skipping drugs for Duchenne muscular dystrophy over safety concerns, in ...

The researchers will explore non-viral options for delivering gene therapy to potential DMD patients, specifically ...

People hit the road for a short race Saturday to support research into Duchenne muscular dystrophy. The GR .1K was held ...

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is ...

PTC Therapeutics' NDA resubmission of Translarna was based on data from Study 041 and the long-term STRIDE registry. No PDUFA action date was provided for regulators.

Sarepta Therapeutics is stopping development of vesleteplirsen, an experimental exon 51-skipping therapy for Duchenne muscular dystrophy patients with certain mutations. It's also halting work on all ...

People with Myotonic Dystrophy Type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose ...

Researchers created a mouse model to study liver symptoms of myotonic dystrophy type 1, revealing how toxic RNA in the liver ...

1 The treatment landscape has not meaningfully changed in decades, despite the fact that currently available therapies ... candidates including SGT-003 for the treatment of Duchenne muscular dystrophy ...