文献
J-GLOBAL ID:202202268235445691
整理番号:22A0233606
2型および非歩行型3型脊髄性筋萎縮症(SUNFISH)における1日1回リスジプラムの安全性および有効性: 第3相二重盲検無作為化プラセボ対照試験【JST・京大機械翻訳】
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
著者 (31件):
Mercuri Eugenio
(Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy)
,
Deconinck Nicolas
(Neuromuscular Reference Center, UZ Gent, Ghent, Belgium)
,
Deconinck Nicolas
(Neuromuscular Reference Center and Paediatric Neurology, Queen Fabiola Children’s University Hospital, Universite Libre de Bruxelles, Brussels, Belgium)
,
Mazzone Elena S
(Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy)
,
Nascimento Andres
(Neuromuscular Unit, Neuropaediatrics Department, Hospital Sant Joan de Deu, Fundacion Sant Joan de Deu, CIBERER - ISC III, Barcelona, Spain)
,
Oskoui Maryam
(Department of Pediatrics and Department of Neurology and Neurosurgery, McGill University, Montreal, QC, Canada)
,
Saito Kayoko
(Institute of Medical Genetics, Tokyo Women’s Medical University, Tokyo, Japan)
,
Vuillerot Carole
(Service de Reeducation Pediatrique Infantile “L’Escale”, Hopital Femme Mere Enfant, CHU-Lyon, Bron, France)
,
Vuillerot Carole
(Neuromyogen Institute, CNRS UMR 5310 - INSERM U1217 Universite de Lyon, Lyon, France)
,
Baranello Giovanni
(The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, Great Ormond Street NHS Trust, London, UK)
,
Baranello Giovanni
(Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy)
,
Boespflug-Tanguy Odile
(I-Motion, Institut de Myologie, APHP, Hopital Armand Trousseau, Paris, France)
,
Boespflug-Tanguy Odile
(NeuroDiderot, UMR 1141, Universite de Paris, Paris, France)
,
Goemans Nathalie
(Neuromuscular Reference Centre, Department of Paediatrics and Child Neurology, University Hospitals Leuven, Leuven, Belgium)
,
Kirschner Janbernd
(Department of Neuropediatrics and Muscle Disorders, Medical Center-University of Freiburg, Freiburg, Germany)
,
Kirschner Janbernd
(Division of Neuropediatrics, Faculty of Medicine, University Hospital Bonn, Bonn, Germany)
,
Kostera-Pruszczyk Anna
(Department of Neurology, Medical University of Warsaw, Warsaw, Poland)
,
Servais Laurent
(I-Motion, Institut de Myologie, APHP, Hopital Armand Trousseau, Paris, France)
,
Servais Laurent
(MDUK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford, UK)
,
Servais Laurent
(Reference Center for Neuromuscular Disease, Centre Hospitalier Regional de La Citadelle, Liege, Belgium)
,
Gerber Marianne
(F Hoffmann-La Roche, Basel, Switzerland)
,
Gorni Ksenija
(F Hoffmann-La Roche, Basel, Switzerland)
,
Khwaja Omar
(F Hoffmann-La Roche, Basel, Switzerland)
,
Khwaja Omar
(Voyager Therapeutics, Cambridge, MA, USA)
,
Kletzl Heidemarie
(F Hoffmann-La Roche, Basel, Switzerland)
,
Scalco Renata S
(F Hoffmann-La Roche, Basel, Switzerland)
,
Staunton Hannah
(Roche Products, Welwyn Garden City, UK)
,
Yeung Wai Yin
(Roche Products, Welwyn Garden City, UK)
,
Martin Carmen
(Roche Products, Welwyn Garden City, UK)
,
Fontoura Paulo
(F Hoffmann-La Roche, Basel, Switzerland)
,
Day John W
(Department of Neurology, Stanford University, Palo Alto, CA, USA)
資料名:
Lancet Neurology
(Lancet Neurology)
巻:
21
号:
1
ページ:
42-52
発行年:
2022年
JST資料番号:
W3105A
ISSN:
1474-4422
資料種別:
逐次刊行物 (A)
記事区分:
原著論文
発行国:
オランダ (NLD)
言語:
英語 (EN)