Muscular Dystrophy News

1 week ago | musculardystrophynews.com | Marisa Wexler

The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August. The FDA agreed this year to review an application to approve deramiocel for DMD cardiomyopathy, or disease of the heart muscle. The agency granted the application priority review, shortening the review time to six months down from the usual 10 months, with a decision expected by Aug. 31.

2 weeks ago | musculardystrophynews.com | Patricia Inacio

People with myotonic dystrophy (DM) use more healthcare resources and face higher associated costs than patients without the disease, according to a U.S. study that analyzed a large database of insurance claims. After diagnosis, DM patients are more likely to be hospitalized and visit an emergency department, researchers found. Factors contributing to higher care costs include the need for gait aids or respiratory support devices.

3 weeks ago | musculardystrophynews.com | Andrea Lobo

CureDuchenne will host its annual Futures National Conference May 22-25 in San Antonio, as part of an effort to provide education and offer connections to members of the Duchenne and Becker muscular dystrophy communities. Under the theme “Together We Thrive,” the event will share information on research and resources for families affected by Duchenne muscular dystrophy. Registration is open and participants can attend online or in person.

3 weeks ago | musculardystrophynews.com | Marisa Wexler

It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and Drug Administration (FDA), marking the first time a gene therapy for DMD was authorized in the U.S.Since its launch, the response to Elevidys has been “overwhelmingly positive,” James Richardson, MD, Sarepta’s chief medical officer, told Muscular Dystrophy News Today in a written Q&A.

4 weeks ago | musculardystrophynews.com | Marisa Wexler |Patrick Moeschen |Lindsey Shapiro

The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies already in clinical testing for other types of LGMD.