{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,12,20]],"date-time":"2025-12-20T21:57:55Z","timestamp":1766267875804},"reference-count":0,"publisher":"The Medical Letter, Inc.","issue":"1686","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Med Lett Drugs Ther"],"abstract":"<jats:p>Delandistrogene moxeparvovec-rokl (Elevidys \u2013 Sarepta), an adeno-associated virus (AAV) vector-based gene therapy, has received accelerated approval from the FDA for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene. It is the first gene therapy to be approved in the US for treatment of DMD. Accelerated approval was based on expression of microdystrophin in skeletal muscle, a surrogate endpoint.<\/jats:p>","DOI":"10.58347\/tml.2023.1686d","type":"journal-article","created":{"date-parts":[[2023,9,26]],"date-time":"2023-09-26T16:26:39Z","timestamp":1695745599000},"page":"159-160","source":"Crossref","is-referenced-by-count":4,"title":["Delandistrogene Moxeparvovec (Elevidys) for Duchenne Muscular Dystrophy"],"prefix":"10.58347","volume":"65","author":[{"name":"Editors from The Medical Letter","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"37151","published-online":{"date-parts":[[2023,10,2]]},"container-title":["The Medical Letter on Drugs and Therapeutics"],"original-title":[],"deposited":{"date-parts":[[2023,9,26]],"date-time":"2023-09-26T16:26:39Z","timestamp":1695745599000},"score":1,"resource":{"primary":{"URL":"https:\/\/secure.medicalletter.org\/TML-article-1686d"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,10,2]]},"references-count":0,"journal-issue":{"issue":"1686","published-online":{"date-parts":[[2023,10,2]]}},"URL":"https:\/\/doi.org\/10.58347\/tml.2023.1686d","relation":{},"ISSN":["1523-2859"],"issn-type":[{"value":"1523-2859","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,10,2]]}}}