Perry B Shieh, Wendy Hughes, Marie Wood, Alan H Beggs, Michael W Lawlor, Julie Coats, Fatbardha Varfaj, Robert J Graham, Nancy L Kuntz, James J Dowling, Wolfgang Müller-Felber, Carsten G Bönnemann, Ana Buj Bello, Laurent Servais, Vicky MacBean, Francesco Muntoni, A Reghan Foley, Astrid Blaschek, Emma S James, Andreea Seferian, Lindsay N Alfano, Tina Duong, Mojtaba Noursalehi, Weston Miller, Jun Lee, Suyash Prasad, Salvador Rico
{"title":"儿童x连锁肌小管肌病的基因治疗：对ASPIRO研究发表的简单语言总结。","authors":"Perry B Shieh, Wendy Hughes, Marie Wood, Alan H Beggs, Michael W Lawlor, Julie Coats, Fatbardha Varfaj, Robert J Graham, Nancy L Kuntz, James J Dowling, Wolfgang Müller-Felber, Carsten G Bönnemann, Ana Buj Bello, Laurent Servais, Vicky MacBean, Francesco Muntoni, A Reghan Foley, Astrid Blaschek, Emma S James, Andreea Seferian, Lindsay N Alfano, Tina Duong, Mojtaba Noursalehi, Weston Miller, Jun Lee, Suyash Prasad, Salvador Rico","doi":"10.1177/26330040251362885","DOIUrl":null,"url":null,"abstract":"<p><p>What is this summary about? This summary describes the results of a research study (clinical trial) called ASPIRO that was published in the <i>Lancet Neurology</i> in 2023. This study looked at an investigational gene therapy called <b>resamirigene bilparvovec</b> (also known as AT132) as a possible treatment for children with a disease called X-linked <b>myotubular myopathy</b> (abbreviated as XLMTM).</p>","PeriodicalId":75218,"journal":{"name":"Therapeutic advances in rare disease","volume":"6 ","pages":"26330040251362885"},"PeriodicalIF":0.0000,"publicationDate":"2025-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12449642/pdf/","citationCount":"0","resultStr":"{\"title\":\"Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study.\",\"authors\":\"Perry B Shieh, Wendy Hughes, Marie Wood, Alan H Beggs, Michael W Lawlor, Julie Coats, Fatbardha Varfaj, Robert J Graham, Nancy L Kuntz, James J Dowling, Wolfgang Müller-Felber, Carsten G Bönnemann, Ana Buj Bello, Laurent Servais, Vicky MacBean, Francesco Muntoni, A Reghan Foley, Astrid Blaschek, Emma S James, Andreea Seferian, Lindsay N Alfano, Tina Duong, Mojtaba Noursalehi, Weston Miller, Jun Lee, Suyash Prasad, Salvador Rico\",\"doi\":\"10.1177/26330040251362885\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>What is this summary about? This summary describes the results of a research study (clinical trial) called ASPIRO that was published in the <i>Lancet Neurology</i> in 2023. This study looked at an investigational gene therapy called <b>resamirigene bilparvovec</b> (also known as AT132) as a possible treatment for children with a disease called X-linked <b>myotubular myopathy</b> (abbreviated as XLMTM).</p>\",\"PeriodicalId\":75218,\"journal\":{\"name\":\"Therapeutic advances in rare disease\",\"volume\":\"6 \",\"pages\":\"26330040251362885\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2025-09-18\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12449642/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Therapeutic advances in rare disease\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.1177/26330040251362885\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Therapeutic advances in rare disease","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.1177/26330040251362885","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"","JCRName":"","Score":null,"Total":0}
Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study.
What is this summary about? This summary describes the results of a research study (clinical trial) called ASPIRO that was published in the Lancet Neurology in 2023. This study looked at an investigational gene therapy called resamirigene bilparvovec (also known as AT132) as a possible treatment for children with a disease called X-linked myotubular myopathy (abbreviated as XLMTM).
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