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

Springer Science and Business Media LLC - Tập 12 Số 2 - Trang 543-557 - 2023
Claudia A. Chiriboga1, Claudio Bruno2, Tina Duong3, Dirk Fischer4, Eugenio Mercuri5, Janbernd Kirschner6, Anna Kostera‐Pruszczyk7,8, Birgit Jaber9, Ksenija Gorni10, Heidemarie Kletzl11, Imogen Carruthers12, Carmen Martín12, Francis Warren12, Renata S. Scalco13, Kathryn R. Wagner13, Francesco Muntoni14
1Department of Neurology, Columbia University Irving Medical Center, 180 Fort Washington Avenue # 552, New York, NY, 10032-3791, USA. [email protected].
2Centre of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, and Department of Neuroscience, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health-DINOGMI, University of Genoa, Genoa, Italy.
3Department of Neurology, Stanford University, Palo Alto, CA, USA
4Division of Neuropediatrics, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
5Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy
6Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany
7Department of Neurology, Medical University of Warsaw, Warsaw, Poland
8ERN EURO-NMD, Warsaw, Poland.
9Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland
10PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland
11Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland
12Roche Products Ltd, Welwyn Garden City, UK
13Product Development Neuroscience, F. Hoffmann-La Roche Ltd, Basel, Switzerland
14The Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London and Great Ormond Street Hospital Trust, London, UK

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Springer Science and Business Media LLC

Tập 12 Số 2

543-557

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