{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T07:49:19Z","timestamp":1753861759973,"version":"3.41.2"},"reference-count":3,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2024,6,7]],"date-time":"2024-06-07T00:00:00Z","timestamp":1717718400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J of Med Genetics Pt A"],"published-print":{"date-parts":[[2024,7]]},"DOI":"10.1002\/ajmg.a.63276","type":"journal-article","created":{"date-parts":[[2024,6,7]],"date-time":"2024-06-07T15:28:15Z","timestamp":1717774095000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["New Gene Therapy Changes Treatment Landscape for Metachromatic Leukodystrophy"],"prefix":"10.1002","volume":"194","member":"311","published-online":{"date-parts":[[2024,6,7]]},"reference":[{"key":"e_1_2_2_2_1","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1186\/s13023-023-02814-2","article-title":"A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel","volume":"18","author":"Armstrong N","year":"2023","journal-title":"Orphanet J Rare Dis"},{"key":"e_1_2_2_3_1","unstructured":"FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy. Press release March 18 2024. Available at:https:\/\/www.fda.gov\/news\u2010events\/press\u2010announcements\/fda\u2010approves\u2010first\u2010gene\u2010therapy\u2010children\u2010metachromatic\u2010leukodystrophy. Accessed March 18 2024."},{"key":"e_1_2_2_4_1","doi-asserted-by":"crossref","unstructured":"OrchardPJ GuptaAO BraunJ et al.Compassionate use of lentiviral gene therapy for metachromatic leukodystrophy. WORLDSymposium presentation February 7 2024.","DOI":"10.1016\/j.ymgme.2023.107989"}],"container-title":["American Journal of Medical Genetics Part A"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajmg.a.63276","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,6,7]],"date-time":"2024-06-07T15:29:21Z","timestamp":1717774161000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajmg.a.63276"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,6,7]]},"references-count":3,"journal-issue":{"issue":"7","published-print":{"date-parts":[[2024,7]]}},"alternative-id":["10.1002\/ajmg.a.63276"],"URL":"https:\/\/doi.org\/10.1002\/ajmg.a.63276","archive":["Portico"],"relation":{},"ISSN":["1552-4825","1552-4833"],"issn-type":[{"type":"print","value":"1552-4825"},{"type":"electronic","value":"1552-4833"}],"subject":[],"published":{"date-parts":[[2024,6,7]]},"assertion":[{"value":"2023-07-22","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2023-11-05","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-06-07","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e63276"}}