Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study

被引：0
|
作者：
Claudia A. Chiriboga
Claudio Bruno
Tina Duong
Dirk Fischer
Eugenio Mercuri
Janbernd Kirschner
Anna Kostera-Pruszczyk
Birgit Jaber
Ksenija Gorni
Heidemarie Kletzl
Imogen Carruthers
Carmen Martin
Francis Warren
Renata S. Scalco
Kathryn R. Wagner
Francesco Muntoni
机构：
[1] Columbia University Irving Medical Center,Department of Neurology
[2] University of Genoa,Centre of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, and Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health
[3] Stanford University,DINOGMI
[4] University Children’s Hospital Basel,Department of Neurology
[5] University of Basel,Division of Neuropediatrics
[6] Fondazione Policlinico Gemelli IRCCS,Pediatric Neurology Institute, Catholic University and Nemo Pediatrico
[7] Medical Center-University of Freiburg,Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine
[8] Medical University of Warsaw,Department of Neurology
[9] ERN EURO-NMD,Roche Pharmaceutical Research and Early Development
[10] Pharma Development,Product Development Neuroscience
[11] Safety,The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre
[12] F. Hoffmann-La Roche Ltd,undefined
[13] PDMA Neuroscience and Rare Disease,undefined
[14] F. Hoffmann-La Roche Ltd,undefined
[15] Roche Innovation Center Basel,undefined
[16] Roche Products Ltd,undefined
[17] F. Hoffmann-La Roche Ltd,undefined
[18] Great Ormond Street Institute of Child Health University College London,undefined
[19] and Great Ormond Street Hospital Trust,undefined
来源：
Neurology and Therapy | 2023年 / 12卷
关键词：
Evrysdi; Pharmacodynamics; Risdiplam; Safety; Spinal muscular atrophy;
D O I：
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中图分类号：
学科分类号：
摘要：
引用
收藏
页码：543 / 557
页数：14
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