{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,10]],"date-time":"2026-09-10T15:54:24Z","timestamp":1789055664605,"version":"build-2803163510"},"reference-count":0,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"name":"Astellas Gene Therapies"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Therapeutic Advances in Rare Disease"],"published-print":{"date-parts":[[2025,9]]},"abstract":"<jats:p>\n                    <jats:bold>What is this summary about?<\/jats:bold>\n                  <\/jats:p>\n                  <jats:p>\n                    This summary describes the results of a research study (clinical trial) called ASPIRO that was published in the\n                    <jats:italic>Lancet Neurology<\/jats:italic>\n                    in 2023. This study looked at an investigational gene therapy called\n                    <jats:bold>resamirigene bilparvovec<\/jats:bold>\n                    (also known as AT132) as a possible treatment for children with a disease called X-linked\n                    <jats:bold>myotubular myopathy<\/jats:bold>\n                    (abbreviated as XLMTM).\n                  <\/jats:p>","DOI":"10.1177\/26330040251362885","type":"journal-article","created":{"date-parts":[[2025,9,19]],"date-time":"2025-09-19T06:11:11Z","timestamp":1758262271000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":1,"title":["Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study"],"prefix":"10.1177","volume":"6","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7145-7663","authenticated-orcid":false,"given":"Perry B.","family":"Shieh","sequence":"first","affiliation":[{"name":"Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wendy","family":"Hughes","sequence":"additional","affiliation":[{"name":"Myotubular Trust, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-1665-3004","authenticated-orcid":false,"given":"Marie","family":"Wood","sequence":"additional","affiliation":[{"name":"MTM-CNM Family Connection, Methuen, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alan H.","family":"Beggs","sequence":"additional","affiliation":[{"name":"Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children\u2019s Hospital, Harvard Medical School, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0731-3950","authenticated-orcid":false,"given":"Michael W.","family":"Lawlor","sequence":"additional","affiliation":[{"name":"Department of Pathology and Laboratory Medicine, Medical College of Wisconsin, and Diverge Translational Science Laboratory, Milwaukee, WI, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie","family":"Coats","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fatbardha","family":"Varfaj","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert J.","family":"Graham","sequence":"additional","affiliation":[{"name":"Division of Critical Care Medicine, Boston Children\u2019s Hospital, Harvard Medical School, Boston, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy L.","family":"Kuntz","sequence":"additional","affiliation":[{"name":"Division of Neurology, Ann &amp; Robert H Lurie Children\u2019s Hospital of Chicago, Chicago, IL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James J.","family":"Dowling","sequence":"additional","affiliation":[{"name":"Division of Neurology, The Hospital for Sick Children, Toronto, ON, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wolfgang","family":"M\u00fcller-Felber","sequence":"additional","affiliation":[{"name":"Department of Paediatric Neurology and Developmental Medicine, Hauner Children\u2019s Hospital, Ludwig Maximilian University of Munich, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carsten G.","family":"B\u00f6nnemann","sequence":"additional","affiliation":[{"name":"Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Buj Bello","sequence":"additional","affiliation":[{"name":"G\u00e9n\u00e9thon, Evry, France, and Universit\u00e9 Paris-Saclay\/Universit\u00e9 Evry, INSERM, G\u00e9n\u00e9thon, INTEGRARE Research Unit UMR_S951, Evry, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurent","family":"Servais","sequence":"additional","affiliation":[{"name":"Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0268-2693","authenticated-orcid":false,"given":"Vicky","family":"MacBean","sequence":"additional","affiliation":[{"name":"Department of Health Sciences, Brunel University of London, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Muntoni","sequence":"additional","affiliation":[{"name":"NIHR, Great Ormond Street Hospital, Biomedical Research Centre, University College London Institute of Child Health, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A. Reghan","family":"Foley","sequence":"additional","affiliation":[{"name":"Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Astrid","family":"Blaschek","sequence":"additional","affiliation":[{"name":"Department of Paediatric Neurology and Developmental Medicine, Hauner Children\u2019s Hospital, Ludwig Maximilian University of Munich, Munich, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma S.","family":"James","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andreea","family":"Seferian","sequence":"additional","affiliation":[{"name":"I-Motion, H\u00f4pital Armand Trousseau, Paris, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lindsay N.","family":"Alfano","sequence":"additional","affiliation":[{"name":"Abigail Wexner Research Institute, Nationwide Children\u2019s Hospital, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tina","family":"Duong","sequence":"additional","affiliation":[{"name":"Department of Neurology, Stanford University, Palo Alto, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mojtaba","family":"Noursalehi","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Weston","family":"Miller","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jun","family":"Lee","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suyash","family":"Prasad","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salvador","family":"Rico","sequence":"additional","affiliation":[{"name":"Astellas Gene Therapies, San Francisco, CA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2025,9,18]]},"container-title":["Therapeutic Advances in Rare Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/26330040251362885","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/26330040251362885","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/26330040251362885","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T01:10:49Z","timestamp":1777597849000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/26330040251362885"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9]]},"references-count":0,"alternative-id":["10.1177\/26330040251362885"],"URL":"https:\/\/doi.org\/10.1177\/26330040251362885","relation":{},"ISSN":["2633-0040","2633-0040"],"issn-type":[{"value":"2633-0040","type":"print"},{"value":"2633-0040","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,9]]},"article-number":"26330040251362885"}}