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

Chiriboga, Claudia A; Bruno, Claudio; Duong, Tina; Fischer, Dirk; Mercuri, Eugenio; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Jaber, Birgit; Gorni, Ksenija; Kletzl, Heidemarie; Carruthers, Imogen; Martin, Carmen; Warren, Francis; Scalco, Renata S; Wagner, Kathryn R; Muntoni, Francesco

Chiriboga, Claudia A; Bruno, Claudio; Duong, Tina; Fischer, Dirk; Mercuri, Eugenio; Kirschner, Janbernd; Kostera-Pruszczyk, Anna; Jaber, Birgit; Gorni, Ksenija; Kletzl, Heidemarie; Carruthers, Imogen; Martin, Carmen; Warren, Francis; Scalco, Renata S; Wagner, Kathryn R; Muntoni, Francesco.

Afiliación

Chiriboga CA; Department of Neurology, Columbia University Irving Medical Center, 180 Fort Washington Avenue # 552, New York, NY, 10032-3791, USA. cac3@cumc.columbia.edu.
Bruno C; Centre 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.
Duong T; Department of Neurology, Stanford University, Palo Alto, CA, USA.
Fischer D; Division of Neuropediatrics, University Children's Hospital Basel, University of Basel, Basel, Switzerland.
Mercuri E; Pediatric Neurology Institute, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome, Italy.
Kirschner J; Department of Neuropediatrics and Muscle Disorders, Faculty of Medicine, Medical Center-University of Freiburg, Freiburg, Germany.
Kostera-Pruszczyk A; Department of Neurology, Medical University of Warsaw, Warsaw, Poland.
Jaber B; ERN EURO-NMD, Warsaw, Poland.
Gorni K; Pharma Development, Safety, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Kletzl H; PDMA Neuroscience and Rare Disease, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Carruthers I; Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland.
Martin C; Roche Products Ltd, Welwyn Garden City, UK.
Warren F; Roche Products Ltd, Welwyn Garden City, UK.
Scalco RS; Roche Products Ltd, Welwyn Garden City, UK.
Wagner KR; Product Development Neuroscience, F. Hoffmann-La Roche Ltd, Basel, Switzerland.
Muntoni F; Product Development Neuroscience, F. Hoffmann-La Roche Ltd, Basel, Switzerland.