{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,12]],"date-time":"2026-06-12T21:23:56Z","timestamp":1781299436751,"version":"3.54.1"},"reference-count":6,"publisher":"American Medical Association (AMA)","issue":"13","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA"],"published-print":{"date-parts":[[2024,4,2]]},"abstract":"<jats:p>This study estimates public and private spending on genetically targeted treatments for Duchenne muscular dystrophy during years in which the drugs were marketed without completed confirmatory studies.<\/jats:p>","DOI":"10.1001\/jama.2024.2776","type":"journal-article","created":{"date-parts":[[2024,3,11]],"date-time":"2024-03-11T15:01:06Z","timestamp":1710169266000},"page":"1151","source":"Crossref","is-referenced-by-count":11,"title":["Spending on Targeted Therapies for Duchenne Muscular Dystrophy"],"prefix":"10.1001","volume":"331","author":[{"given":"Liam","family":"Bendicksen","sequence":"first","affiliation":[{"name":"Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women\u2019s Hospital, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron S.","family":"Kesselheim","sequence":"additional","affiliation":[{"name":"Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women\u2019s Hospital, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin N.","family":"Rome","sequence":"additional","affiliation":[{"name":"Division of Pharmacoepidemiology and Pharmacoeconomics, Brigham and Women\u2019s Hospital, Boston, Massachusetts"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"10","reference":[{"issue":"9","key":"jld240012r1","doi-asserted-by":"publisher","first-page":"1251","DOI":"10.7326\/M23-1073","article-title":"The regulatory repercussions of approving muscular dystrophy medications on the basis of limited evidence.","volume":"176","author":"Bendicksen","year":"2023","journal-title":"Ann Intern Med"},{"issue":"22","key":"jld240012r4","doi-asserted-by":"publisher","first-page":"2357","DOI":"10.1001\/jama.2016.16437","article-title":"Approving a problematic muscular dystrophy drug: implications for FDA policy.","volume":"316","author":"Kesselheim","year":"2016","journal-title":"JAMA"},{"issue":"3","key":"jld240012r5","doi-asserted-by":"publisher","DOI":"10.1001\/jamahealthforum.2023.0217","article-title":"Timing of confirmatory trials for drugs granted accelerated approval based on surrogate measures from 2012 to 2021.","volume":"4","author":"Deshmukh","year":"2023","journal-title":"JAMA Health Forum"},{"key":"jld240012r2","unstructured":"Sarepta Therapeutics announces topline results from EMBARK, a global pivotal study of ELEVIDYS gene therapy for Duchenne muscular dystrophy. Press release. Sarepta Therapeutics. October 30, 2023. Accessed February 5, 2024. https:\/\/investorrelations.sarepta.com\/news-releases\/news-release-details\/sarepta-therapeutics-announces-topline-results-embark-global-0"},{"key":"jld240012r3","unstructured":"National Institutes of Health. Duchenne muscular dystrophy. Accessed January 16, 2024. https:\/\/rarediseases.info.nih.gov\/diseases\/6291\/duchenne-muscular-dystrophy"},{"key":"jld240012r6","unstructured":"Bayer? M. The future of accelerated approval for FDA\u2019s Marks? more rare disease drugs and no \u201changing chads.\u201d Fierce Biotech. July 28, 2023. Accessed December 6, 2023. https:\/\/www.fiercebiotech.com\/biotech\/peter-marks-says-fda-now-has-better-sense-rare-disease-biomarkers-will-more-accelerated"}],"container-title":["JAMA"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/jamanetwork.com\/journals\/jama\/articlepdf\/2816420\/jama_bendicksen_2024_ld_240012_1711564200.25351.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,4,2]],"date-time":"2024-04-02T15:26:32Z","timestamp":1712071592000},"score":1,"resource":{"primary":{"URL":"https:\/\/jamanetwork.com\/journals\/jama\/fullarticle\/2816420"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4,2]]},"references-count":6,"journal-issue":{"issue":"13","published-print":{"date-parts":[[2024,4,2]]}},"URL":"https:\/\/doi.org\/10.1001\/jama.2024.2776","relation":{},"ISSN":["0098-7484"],"issn-type":[{"value":"0098-7484","type":"print"}],"subject":[],"published":{"date-parts":[[2024,4,2]]}}}