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Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.

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Title: Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Authors: Lawlor MW; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA; Diverge Translational Science Laboratory, Milwaukee, WI, 53204, USA. Electronic address: mlawlor@divergetsl.com., Schoser B; Friedrich-Baur-Institute, Department of Neurology, Ludwig Maximilian University of Munich, 80336, Germany., Margeta M; Department of Pathology, University of California San Francisco, San Francisco, CA, 94143, USA., Sewry CA; Wolfson Centre of Inherited Neuromuscular Disorders, RJAH Orthopaedic Hospital, Oswestry, SY10 7AG, UK; Dubowitz Neuromuscular Centre, UCL Institute of Child Health and Great Ormond Street Hospital for Children, 30 Guilford Street, London, WC1N 1EH, UK., Jones KA; Department of Pathology, Duke University School of Medicine, Durham, NC, 27710, USA., Shieh PB; Department of Neurology, University of California Los Angeles School of Medicine, Los Angeles, CA, 90095, USA., Kuntz NL; Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, 60611, USA., Smith BK; Department of Physical Therapy, University of Florida, Gainesville, FL, 32610-0154, USA., Dowling JJ; Hospital for Sick Children, Toronto, ON, M5G 1X8, Canada., Müller-Felber W; Dr. von Hauner Children's Hospital, Klinikum der Universität München, 80337, Munich, Germany., Bönnemann CG; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, 20892-1477, USA., Seferian AM; I-Motion, Hôpital Armand Trousseau, 75571, Paris, France., Blaschek A; Dr. von Hauner Children's Hospital, Klinikum der Universität München, 80337, Munich, Germany., Neuhaus S; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, 20892-1477, USA., Foley AR; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, 20892-1477, USA., Saade DN; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, 20892-1477, USA., Tsuchiya E; Hospital for Sick Children, Toronto, ON, M5G 1X8, Canada., Qasim UR; Department of Neurology, University of California Los Angeles School of Medicine, Los Angeles, CA, 90095, USA., Beatka M; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA; Diverge Translational Science Laboratory, Milwaukee, WI, 53204, USA., Prom MJ; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA; Diverge Translational Science Laboratory, Milwaukee, WI, 53204, USA., Ott E; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA; Diverge Translational Science Laboratory, Milwaukee, WI, 53204, USA., Danielson S; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA., Krakau P; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA; Diverge Translational Science Laboratory, Milwaukee, WI, 53204, USA., Kumar SN; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA., Meng H; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA; Diverge Translational Science Laboratory, Milwaukee, WI, 53204, USA., Vanden Avond M; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA., Wells C; Medical College of Wisconsin, Department of Pathology and Laboratory Medicine, Milwaukee, WI, 53226, USA., Gordish-Dressman H; Children's National Hospital and George Washington University School of Medicine and Health Sciences Department of Pediatrics, Washington, DC, 20037, USA., Beggs AH; Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, 02115, USA., Christensen S; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Conner E; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., James ES; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Lee J; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Sadhu C; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Miller W; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Sepulveda B; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Varfaj F; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Prasad S; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA., Rico S; Formerly of Astellas Gene Therapies (formerly Audentes Therapeutics, Inc.), San Francisco, CA, 94108, USA.
Source: EBioMedicine [EBioMedicine] 2024 Jan; Vol. 99, pp. 104894. Date of Electronic Publication: 2023 Dec 12.
Publication Type: Journal Article
Journal Info: Publisher: Elsevier B.V Country of Publication: Netherlands NLM ID: 101647039 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2352-3964 (Electronic) Linking ISSN: 23523964 NLM ISO Abbreviation: EBioMedicine Subsets: MEDLINE
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  Data: Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
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