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JEWELFISH: Safety and Pharmacodynamic Data in Non-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (RG7916) (772)

Year: 2020

Type: article

Source: Neurology

DOI: 10.1212/wnl.94.15_supplement.772

Authors: Claudia Chiriboga, Claudio Bruno, John W. Day, Tina Duong, Dirk Fischer, Janbernd Kirschner, Francesco Muntoni, Sabine Fuerst-Recktenwald, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Francis Warren, Wai Yin Yeung, Eugenio Mercuri

Institutions: Columbia University Irving Medical Center, Istituto Giannina Gaslini, Stanford University, University of Basel, University Children’s Hospital Basel, University Hospital Bonn, University College London, F. Hoffmann-La Roche AG

Language: English

Topic: Neurogenetic and Muscular Disorders Research

Subfield: Genetics

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4389448581

FWCI: 0.257

Cites:

Cited by: 3

Related to: 10