therapy

FDA extends approval of first gene therapy for rare form of muscular dystrophy

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CNN – The US Food and Drug Administration has given the green light for the first gene therapy to treat a rare form of muscular dystrophy to be used in most people who have the disease and a certain genetic mutation. Last year, the drug – Elevidys, from the biotech company Sarepta Therapeutics – was … Read more

FDA expands approval of gene therapy for patients with Duchenne muscular dystrophy

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For immediate release: June 20, 2024 Today, the US Food and Drug Administration extended the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a confirmed mutation in DMD gene. Elevidys was previously approved … Read more