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FDA expands approval of first gene therapy for rare form of muscular …
Jun 20, 2024 · The US 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...
FDA approves $3.2 million gene therapy for rare muscular dystrophy …
Jun 22, 2023 · The US Food and Drug Administration has approved the first gene therapy to treat a rare and devastating muscle disease but limited the approval to kids ages 4 and 5 based on available evidence ...
Muscular dystrophy patients get first gene therapy - NPR
Jun 22, 2023 · Sarepta's muscular dystrophy treatment is the first gene therapy approved under the program. The disease, which almost exclusively affects boys, destroys muscles.
FDA advisers narrowly vote in favor of experimental gene therapy …
May 12, 2023 · SRP-9001, an experimental gene therapy that aims to slow or stop the progression of Duchenne muscular dystrophy, is being considered for accelerated approval by the FDA.
FDA Expands Approval of Gene Therapy for Patients with …
Jun 20, 2024 · The FDA expanded the approval of Elevidys, a gene therapy for the treatment of Duchenne muscular dystrophy for ambulatory and non-ambulatory individuals 4 years of age and older with DMD with a...
FDA Duchenne muscular dystrophy gene therapy : Shots - Health News - NPR
May 12, 2023 · A panel of experts voted 8-6 in favor of Food and Drug Administration approval of the first gene therapy for Duchenne muscular dystrophy, a fatal genetic disease.
New Gene Therapy for Duchenne Muscular Dystrophy
Oct 23, 2024 · Without dystrophin, muscle becomes fragile and easily degenerates. A new single-dose intravenous gene therapy, delandistrogene moxeparvovec, creates “micro-dystrophin” — a scaled-down version of the large gene that encodes dystrophin — …
FDA approves first gene therapy for Duchenne muscular dystrophy ...
Jun 23, 2023 · The FDA has granted accelerated approval to Sarepta’s gene therapy delandistrogene moxeparvovec (Elevidys) for Duchenne muscular dystrophy (DMD). It is the first time the FDA has greenlit a...
‘It’s a vote for hope’: first gene therapy for muscular dystrophy …
Jun 2, 2023 · Update: The US Food and Drug Administration has now approved the first gene therapy for Duchenne muscular dystrophy for children aged four and five. The one-time treatment, named Elevidys, will...