USA - FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy (Catapult's regulatory round-up - June)
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21 février 2024
- doi: 10.58079/vvn9
info:eu-repo/semantics/openAccess
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adelage, « USA - FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy (Catapult's regulatory round-up - June) », ELSIBI, ID : 10.58079/vvn9
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On the 22nd of June 2023 the FDA approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy. Elevidys, is a recombinant gene therapy designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin, a shortened protein (138 kDa, compared to the 427 kDa...