作者：
Perry B,Shieh [1]
;
Nancy L,Kuntz [2]
;
James J,Dowling [3]
;
Wolfgang,Müller-Felber [4]
;
Carsten G,Bönnemann [5]
;
Andreea M,Seferian [6]
;
Laurent,Servais [7]
;
Barbara K,Smith [8]
;
Francesco,Muntoni [9]
;
Astrid,Blaschek [4]
;
A Reghan,Foley [5]
;
Dimah N,Saade [10]
;
Sarah,Neuhaus [5]
;
Lindsay N,Alfano [11]
;
Alan H,Beggs [12]
;
Ana,Buj-Bello [13]
;
Martin K,Childers [14]
;
Tina,Duong [15]
;
Robert J,Graham [16]
;
Minal,Jain [17]
;
Julie,Coats [18]
;
Vicky,MacBean [19]
;
Emma S,James [18]
;
Jun,Lee [18]
;
Fulvio,Mavilio [20]
;
Weston,Miller [18]
;
Fatbardha,Varfaj [18]
;
Michael,Murtagh [18]
;
Cong,Han [21]
;
Mojtaba,Noursalehi [18]
;
Michael W,Lawlor [22]
;
Suyash,Prasad [18]
;
Salvador,Rico [18]
作者单位：
Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA. Electronic address: pshieh@mednet.ucla.edu.
[1]
Division of Neurology, Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, IL, USA.
[2]
Division of Neurology, The Hospital for Sick Children, Toronto, ON, Canada.
[3]
Department of Paediatric Neurology and Developmental Medicine, Hauner Children's Hospital, Ludwig Maximilian University of Munich, Munich, Germany.
[4]
Neuromuscular and Neurogenetic Disorders of Childhood Section, NINDS, NIH, Bethesda, MD, USA.
[5]
I-Motion, Hôpital Armand Trousseau, Paris, France.
[6]
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, Oxford, UK.
[7]
Department of Physical Therapy, University of Florida, Gainesville, FL, USA.
[8]
NIHR, Great Ormond Street Hospital Biomedical Research Centre, University College London Institute of Child Health, London, UK.
[9]
Division of Neurology, University of Iowa Hospitals and Clinics, Iowa City, IA, USA.
[10]
Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.
[11]
Division of Genetics and Genomics, The Manton Center for Orphan Disease Research, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
[12]
Généthon, Evry, France; Integrare Research Unit UMR_S951, Université Paris-Saclay, Université d'Evry, Inserm, Généthon, Evry, France.
[13]
Department of Rehabilitation Medicine, Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
[14]
Department of Neurology, Stanford University, Palo Alto, CA, USA.
[15]
Division of Critical Care Medicine, Boston Children's Hospital, Harvard Medical School, Boston, MA, USA.
[16]
Rehabilitation Medicine Department, NIH Hatfield Clinical Research Center, Bethesda, MD, USA.
[17]
Astellas Gene Therapies, San Francisco, CA, USA.
[18]
Department of Health Sciences, Brunel University London, London, UK.
[19]
Astellas Gene Therapies, San Francisco, CA, USA; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.
[20]
Astellas Pharma Global Development, Northbrook, IL, USA.
[21]
Department of Pathology and Laboratory Medicine, Medical College of Wisconsin, Milwaukee, WI, USA; Diverge Translational Science Laboratory, Milwaukee, WI, USA.
[22]
DOI
10.1016/S1474-4422(23)00313-7
PMID
37977713
发布时间
2024-03-15
换一批
换一批
