Kids with Duchenne muscular dystrophy and their families deserve the chance to decide their own future.

Sarepta Therapeutics Inc. SRPT shares are down during Thursday’s session following a regulatory update regarding its ...

Sarepta Therapeutics says the FDA has agreed to review a regulatory package for Amondys 45 and Vyondys 53 after they failed a ...

Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to disease-related loss of muscle and movement, adverse effects of ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced the ...

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...

The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 years and ...

This week saw both Regenxbio and Solid Bio report new data with their gene therapy candidates for Duchenne muscular dystrophy ...

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene ...

INDIANAPOLIS — A local mom is pushing for a rare and deadly disease to be added to Indiana's newborn screening panel. Duchenne muscular dystrophy affects at least 1 in 5,000 male births each year.

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...