首页> Journal of neuromuscular diseases> Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).

Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG).

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第一作者： Richard S,Finkel

第一单位： Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.;Division of Neurology, Nemours Children's Hospital, Orlando, FL, USA.

作者： Richard S,Finkel ^[1] ; Basil T,Darras ^[2] ; Jerry R,Mendell ^[3] ; John W,Day ^[4] ; Nancy L,Kuntz ^[5] ; Anne M,Connolly ^[6] ; Craig M,Zaidman ^[7] ; Thomas O,Crawford ^[8] ; Russell J,Butterfield ^[9] ; Perry B,Shieh ^[10] ; Gihan,Tennekoon ^[11] ; John F,Brandsema ^[11] ; Susan T,Iannaccone ^[12] ; John,Shoffner ^[13] ; Sarah,Kavanagh ^[14] ; Thomas A,Macek ^[14] ; Sitra,Tauscher-Wisniewski ^[14]

作者单位： Center for Experimental Neurotherapeutics, St. Jude Children's Research Hospital, Memphis, TN, USA.;Division of Neurology, Nemours Children's Hospital, Orlando, FL, USA. ^[1] Department of Neurology, Boston Children's Hospital, Boston, MA, USA. ^[2] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.;Department of Pediatrics and Department of Neurology, The Ohio State University,Columbus, OH, USA. ^[3] Department of Neurology, Stanford University Medical Center, Stanford, CA, USA. ^[4] Division of Neurology, Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, IL, USA. ^[5] Center for Gene Therapy, Nationwide Children's Hospital, Columbus, OH, USA.;Department of Pediatrics and Department of Neurology, The Ohio State University,Columbus, OH, USA.;Division of Neurology, Nationwide Children's Hospital, Columbus, OH, USA. ^[6] Department of Neurology, Division of Pediatric Neurology,Washington University School of Medicine, St. Louis, MO, USA. ^[7] Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA. ^[8] Department of Pediatrics, University of Utah Health, Salt Lake City, UT, USA. ^[9] Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA. ^[10] Division of Neurology, Children's Hospital of Philadelphia, Philadelphia, PA, USA. ^[11] Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA. ^[12] Novartis Gene Therapies, Inc., Bannockburn, IL, USA.;Sangamo Therapeutics, Inc., Richmond, CA, USA. ^[13] Novartis Gene Therapies, Inc., Bannockburn, IL, USA. ^[14]

关键词 Adeno-associated virus Hammersmith Functional Motor Scale Expanded clinical trial gene therapy intrathecal administration motor milestones neurodegenerative disorders onasemnogene abeparvovec spinal muscular atrophy vector genomes

医学主题词人类(Humans);儿童脊髓性肌萎缩(Spinal Muscular Atrophies of Childhood);肌萎缩, 脊髓性(Muscular Atrophy, Spinal);运动神经元(Motor Neurons)

DOI 10.3233/JND-221560

PMID 36911944

发布时间 2023-11-23

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