When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...

The first gene therapy that can treat Duchenne muscular dystrophy (DMD) has been approved by the U.S. Food and Drug Administration; it will be marketed as Elevidys (delandistrogene moxeparvovec-rokl) ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

Scotland’s NHS can now offer givinostat to ambulant children with Duchenne muscular dystrophy, marking a step forward in care ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

Dyne Therapeutics plans to submit its next-gen Duchenne muscular dystrophy drug to the FDA after a successful trial.

Today, the U.S. Department of Health and Human Services (HHS) officially added Duchenne muscular dystrophy (Duchenne) to the Recommended Uniform Screening Panel (RUSP), the national list of conditions ...

The donation from Don and Anne Edwards aims to speed research, treatment and diagnosis for children with rare and genetic ...

The FDA cited risk of IV infection and kidney toxicity in its letter. The company's CEO said the FDA issued no prior warning for the two concerns.