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Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Shieh, Perry B; Kuntz, Nancy L; Dowling, James J; Müller-Felber, Wolfgang; Bönnemann, Carsten G; Seferian, Andreea M; Servais, Laurent; Smith, Barbara K; Muntoni, Francesco; Blaschek, Astrid; Foley, A Reghan; Saade, Dimah N; Neuhaus, Sarah; Alfano, Lindsay N; Beggs, Alan H; Buj-Bello, Ana; Childers, Martin K; Duong, Tina; Graham, Robert J; Jain, Minal; Coats, Julie; MacBean, Vicky; James, Emma S; Lee, Jun; Mavilio, Fulvio; Miller, Weston; Varfaj, Fatbardha; Murtagh, Michael; Han, Cong; Noursalehi, Mojtaba; Lawlor, Michael W; Prasad, Suyash; Rico, Salvador.
Afiliación
  • Shieh PB; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA. Electronic address: pshieh@mednet.ucla.edu.
  • 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.
  • Seferian AM; I-Motion, Hôpital Armand Trousseau, Paris, France.
  • Servais L; I-Motion, Hôpital Armand Trousseau, Paris, France; Neuromuscular Reference Center, Department of Pediatrics, University Hospital Liège, University of Liège, Liège, Belgium; Department of Paediatrics, MDUK Oxford Neuromuscular Centre and NIHR Oxford Biomedical Research Centre, University of Oxford, O
  • Smith BK; Department of Physical Therapy, University of Florida, Gainesville, FL, USA.
  • Muntoni F; NIHR, Great Ormond Street Hospital Biomedical Research Centre, University College London Institute of Child Health, London, UK.
  • Blaschek A; Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany.
  • Foley AR; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA.
  • Saade DN; Division of Neurology, University of Iowa Hospitals and Clinics, Iowa City, IA, USA.
  • Neuhaus S; Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA.
  • Alfano LN; Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, 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.
  • Buj-Bello A; Généthon, Evry, France; Integrare Research Unit UMR_S951, Université Paris-Saclay, Université d'Evry, Inserm, Généthon, Evry, France.
  • Childers MK; Department of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
  • Duong T; Department of Neurology, Stanford University, Palo Alto, CA, USA.
  • Graham RJ; Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
  • Jain M; Rehabilitation Medicine Department, NIH Hatfield Clinical Research Center, Bethesda, MD, USA.
  • Coats J; Astellas Gene Therapies, San Francisco, CA, USA.
  • MacBean V; Department of Health Sciences, Brunel University London, London, UK.
  • James ES; Astellas Gene Therapies, San Francisco, CA, USA.
  • Lee J; Astellas Gene Therapies, San Francisco, CA, USA.
  • Mavilio F; Astellas Gene Therapies, San Francisco, CA, USA; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.
  • Miller W; Astellas Gene Therapies, San Francisco, CA, USA.
  • Varfaj F; Astellas Gene Therapies, San Francisco, CA, USA.
  • Murtagh M; Astellas Gene Therapies, San Francisco, CA, USA.
  • Han C; Astellas Pharma Global Development, Northbrook, IL, USA.
  • Noursalehi M; Astellas Gene Therapies, San Francisco, CA, USA.
  • Lawlor MW; Department of Pathology and Laboratory Medicine, Medical College of Wisconsin, Milwaukee, WI, USA; Diverge Translational Science Laboratory, Milwaukee, WI, USA.
  • Prasad S; Astellas Gene Therapies, San Francisco, CA, USA.
  • Rico S; Astellas Gene Therapies, San Francisco, CA, USA.
Lancet Neurol ; 22(12): 1125-1139, 2023 12.
Article en En | MEDLINE | ID: mdl-37977713
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Sepsis / Miopatías Estructurales Congénitas Límite: Child / Child, preschool / Humans / Infant / Male País/Región como asunto: Europa Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2023 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Sepsis / Miopatías Estructurales Congénitas Límite: Child / Child, preschool / Humans / Infant / Male País/Región como asunto: Europa Idioma: En Revista: Lancet Neurol Asunto de la revista: NEUROLOGIA Año: 2023 Tipo del documento: Article