Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study.
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| Title: | Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study. |
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| Authors: | Shieh PB; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA., Hughes W; Myotubular Trust, London, UK., Wood M; MTM-CNM Family Connection, Methuen, MA, USA., Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Lawlor MW; Department of Pathology and Laboratory Medicine, Medical College of Wisconsin, and Diverge Translational Science Laboratory, Milwaukee, WI, USA., Coats J; Astellas Gene Therapies, San Francisco, CA, USA., Varfaj F; Astellas Gene Therapies, San Francisco, CA, USA., Graham RJ; Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA., Kuntz NL; Division of Neurology, Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA., Dowling JJ; Division of Neurology, The Hospital for Sick Children, Toronto, ON, Canada., Müller-Felber W; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany., Bönnemann CG; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA., Buj Bello A; Généthon, Evry, France, and Université Paris-Saclay/Université Evry, INSERM, Généthon, INTEGRARE Research Unit UMR_S951, Evry, France., Servais L; Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK., MacBean V; Department of Health Sciences, Brunel University of London, London, UK., Muntoni F; NIHR, Great Ormond Street Hospital, Biomedical Research Centre, University College London Institute of Child Health, London, UK., Foley AR; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA., Blaschek A; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany., James ES; Astellas Gene Therapies, San Francisco, CA, USA., Seferian A; I-Motion, Hôpital Armand Trousseau, Paris, France., Alfano LN; Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA., Duong T; Department of Neurology, Stanford University, Palo Alto, CA, USA., Noursalehi M; Astellas Gene Therapies, San Francisco, CA, USA., Miller W; Astellas Gene Therapies, San Francisco, CA, USA., Lee J; Astellas Gene Therapies, San Francisco, CA, USA., Prasad S; Astellas Gene Therapies, San Francisco, CA, USA., Rico S; Astellas Gene Therapies, San Francisco, CA, USA. |
| Source: | Therapeutic advances in rare disease [Ther Adv Rare Dis] 2025 Sep 18; Vol. 6, pp. 26330040251362885. Date of Electronic Publication: 2025 Sep 18 (Print Publication: 2025). |
| Publication Type: | Journal Article; Review |
| Journal Info: | Publisher: Sage Country of Publication: England NLM ID: 9918557474706676 Publication Model: eCollection Cited Medium: Internet ISSN: 2633-0040 (Electronic) Linking ISSN: 26330040 NLM ISO Abbreviation: Ther Adv Rare Dis Subsets: PubMed not MEDLINE |
| Database: | MEDLINE Ultimate |
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| ISSN: | 2633-0040 |
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| DOI: | 10.1177/26330040251362885 |
