Friday, August 4, 2023

Gene Therapy for Duchenne Muscular Dystrophy

On June 22, 2023, it was announced that the U.S. Food and Drug Administration approved the first gene therapy for children ages 4 and 5 years old with Duchenne muscular dystrophy (DMD). In Duchenne muscular dystrophy, there are errors in the genes used to make the protein dystrophin. Dystrophin is needed in muscles to protect them as they contract and relax. Children without this protein have very weak muscles, and they continue to worsen over time. DMD is a common disease as 6 in every 100,000 people in Europe and North America have it; it is most commonly seen in males. The gene therapy is a microdystrophin gene that is packed into harmless viruses for delivery to all cells which produces one-third the size of the original protein. 

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