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USA – FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy (Catapult’s regulatory round-up – June)

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 dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. Please find further information here.

Published on the 3rd of July 2023. Read the Regulatory Round-up – June 2023 (catapult.org.uk) following this link.


OpenEdition vous propose de citer ce billet de la manière suivante :
adelage (21 février 2024). USA – FDA Approves First Gene Therapy for Treatment of Certain Patients with Duchenne Muscular Dystrophy (Catapult’s regulatory round-up – June). ELSIBI. Consulté le 15 mars 2026 à l’adresse https://doi.org/10.58079/vvn9


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