Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial

The Lancet Neurology - Tập 22 - Trang 1125-1139 - 2023
Perry B Shieh1, Nancy L Kuntz2, James J Dowling3, Wolfgang Müller-Felber4, Carsten G Bönnemann5, Andreea M Seferian6, Laurent Servais6,7,8, Barbara K Smith9, Francesco Muntoni10, Astrid Blaschek4, A Reghan Foley5, Dimah N Saade11, Sarah Neuhaus5, Lindsay N Alfano12, Alan H Beggs13, Ana Buj-Bello14,15, Martin K Childers16, Tina Duong17, Robert J Graham18, Minal Jain19
1Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA USA
2Division of Neurology, Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA
3Division of Neurology, The Hospital for Sick Children, Toronto, ON, Canada
4Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany
5Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA
6I-Motion, Hôpital Armand Trousseau, Paris, France
7Neuromuscular Reference Center, Department of Pediatrics, University Hospital Liège, University of Liège, Liège, Belgium
8Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK
9Department of Physical Therapy, University of Florida, Gainesville, FL, USA
10NIHR, Great Ormond Street Hospital Biomedical Research Centre, University College London Institute of Child Health, London, UK
11Division of Neurology, University of Iowa Hospitals and Clinics, Iowa City, IA, USA
12Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA
13Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
14Généthon, Evry, France
15Integrare Research Unit UMR_S951, Université Paris-Saclay, Université d’Evry, Inserm, Généthon, Evry, France
16Department of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA
17Department of Neurology, Stanford University, Palo Alto, CA, USA
18Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA
19Rehabilitation Medicine Department, NIH Hatfield Clinical Research Center, Bethesda, MD, USA

Tài liệu tham khảo

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The Lancet Neurology

Tập 22

1125-1139

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