Today, the U.S. Food and Drug Administration approved Elevidys, the first gene therapy for the treatment of pediatric patients 4 through 5 years of age with Duchenne muscular dystrophy …

Oct 23, 2024 · In June 2023, FDA granted accelerated approval to delandistrogene moxeparvovec (Elevidys®), an adeno-associated virus (AAV) gene therapy for Duchenne …

Oct 23, 2024 · A new single-dose intravenous gene therapy, delandistrogene moxeparvovec, creates “micro-dystrophin” — a scaled-down version of the large gene that encodes dystrophin …

This newly approved gene therapy delivers a copy of a gene that encodes a shortened, functional form of dystrophin, the gene that is mutated in DMD patients. Dystrophin is like a shock …

On Thursday, June 22, the U.S. Food and Drug Administration (FDA) approved Elevidys, the first gene therapy for Duchenne muscular dystrophy (DMD), for patients with the disease between …

Jun 20, 2024 · Last year, the drug – Elevidys, from the biotech company Sarepta Therapeutics – was approved to treat only children ages 4 and 5 with Duchenne muscular dystrophy, one of …

Jun 26, 2023 · This gene transfer therapy uses an adeno-associated virus vector to deliver an engineered mini-dystrophin gene to the muscle cells in order to produce dystrophin, an …

Oct 9, 2024 · Delandistrogene moxeparvovec is a single-administration recombinant adeno-associated virus rhesus isolate serotype 74 (rAAVrh74) vector-based gene transfer therapy, …

Jun 26, 2023 · The U.S. Food and Drug Administration's accelerated approval of the first gene therapy for Duchenne muscular dystrophy (DMD) is a major win for the field of gene therapy …

Jun 23, 2023 · Elevidys works by replacing the Duchenne variant of dystrophin with man-made version based on a version found in a patient with a milder form of the disease, called Becker …