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The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative muscle disease, the agency announced June 22.
Duchenne muscular dystrophy, the most common form of the muscle disease, is caused by mutations in the dystrophin gene, which makes a large protein that acts as a shock absorber to keep muscle cells intact.
Patients with Duchenne muscular dystrophy don’t make this shock-absorbing protein and “damage themselves literally every time they contract their muscles,” Douglas Ingram, president and chief executive of Sarepta Therapeutics, the company that makes the newly approved gene therapy, said in a conference call discussing the approval.
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