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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.
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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  Data: Gene therapy for children with X-linked myotubular myopathy: a plain language summary of publication for the ASPIRO study.
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  Data: <searchLink fieldCode="AU" term="%22Shieh+PB%22">Shieh PB</searchLink>; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA.<br /><searchLink fieldCode="AU" term="%22Hughes+W%22">Hughes W</searchLink>; Myotubular Trust, London, UK.<br /><searchLink fieldCode="AU" term="%22Wood+M%22">Wood M</searchLink>; MTM-CNM Family Connection, Methuen, MA, USA.<br /><searchLink fieldCode="AU" term="%22Beggs+AH%22">Beggs AH</searchLink>; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Lawlor+MW%22">Lawlor MW</searchLink>; Department of Pathology and Laboratory Medicine, Medical College of Wisconsin, and Diverge Translational Science Laboratory, Milwaukee, WI, USA.<br /><searchLink fieldCode="AU" term="%22Coats+J%22">Coats J</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Varfaj+F%22">Varfaj F</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Graham+RJ%22">Graham RJ</searchLink>; Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.<br /><searchLink fieldCode="AU" term="%22Kuntz+NL%22">Kuntz NL</searchLink>; Division of Neurology, Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.<br /><searchLink fieldCode="AU" term="%22Dowling+JJ%22">Dowling JJ</searchLink>; Division of Neurology, The Hospital for Sick Children, Toronto, ON, Canada.<br /><searchLink fieldCode="AU" term="%22Müller-Felber+W%22">Müller-Felber W</searchLink>; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22Bönnemann+CG%22">Bönnemann CG</searchLink>; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Buj+Bello+A%22">Buj Bello A</searchLink>; Généthon, Evry, France, and Université Paris-Saclay/Université Evry, INSERM, Généthon, INTEGRARE Research Unit UMR_S951, Evry, France.<br /><searchLink fieldCode="AU" term="%22Servais+L%22">Servais L</searchLink>; Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK.<br /><searchLink fieldCode="AU" term="%22MacBean+V%22">MacBean V</searchLink>; Department of Health Sciences, Brunel University of London, London, UK.<br /><searchLink fieldCode="AU" term="%22Muntoni+F%22">Muntoni F</searchLink>; NIHR, Great Ormond Street Hospital, Biomedical Research Centre, University College London Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Foley+AR%22">Foley AR</searchLink>; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA.<br /><searchLink fieldCode="AU" term="%22Blaschek+A%22">Blaschek A</searchLink>; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany.<br /><searchLink fieldCode="AU" term="%22James+ES%22">James ES</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Seferian+A%22">Seferian A</searchLink>; I-Motion, Hôpital Armand Trousseau, Paris, France.<br /><searchLink fieldCode="AU" term="%22Alfano+LN%22">Alfano LN</searchLink>; Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.<br /><searchLink fieldCode="AU" term="%22Duong+T%22">Duong T</searchLink>; Department of Neurology, Stanford University, Palo Alto, CA, USA.<br /><searchLink fieldCode="AU" term="%22Noursalehi+M%22">Noursalehi M</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Miller+W%22">Miller W</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Lee+J%22">Lee J</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Prasad+S%22">Prasad S</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.<br /><searchLink fieldCode="AU" term="%22Rico+S%22">Rico S</searchLink>; Astellas Gene Therapies, San Francisco, CA, USA.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Sage%22">Sage </searchLink><i>Country of Publication: </i>England <i>NLM ID: </i>9918557474706676 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Internet <i>ISSN: </i>2633-0040 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2226330040%22">26330040 </searchLink><i>NLM ISO Abbreviation: </i>Ther Adv Rare Dis <i>Subsets: </i>PubMed not MEDLINE
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