Please provide your email address to receive an email when new articles are posted on . Ataluren preserved hand-to-mouth function in boys with nonsense mutation Duchenne muscular dystrophy. Ataluren ...

The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

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 ...

The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the risks and benefits of the drug The Muscular Dystrophy Association trusts the decision of the FDA, which weighs the ...

The therapy, to be marketed as Elevidys, is approved for the treatment of ambulatory pediatric patients aged 4 through 5 years with DMD who have a confirmed mutation in the DMD gene. Sarepta ...

The FDA approved oral givinostat (Duvyzat) to treat Duchenne muscular dystrophy (DMD) in patients ages 6 years and older, the agency announced Thursday. Givinostat is a histone deacetylase (HDAC) ...

DUBLIN--(BUSINESS WIRE)--The "Duchenne Muscular Dystrophy Drugs Market by Product Type, Therapeutic Approach, End User, and Region 2023-2028" report has been added to ResearchAndMarkets.com's offering ...

—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 U.S. Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...