Work

JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory Efficacy Data in Non-Treatment-Naïve Patients with Spinal Muscular Atrophy (SMA) Receiving Treatment with Risdiplam (P7-9.004)

Year: 2023

Type: article

Source: Neurology

DOI: 10.1212/wnl.0000000000203609

Authors: Claudia Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Janbernd Kirschner, Mariacristina Scoto, Eugenio Mercuri, Marianne Gerber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Teresa Gidaro, Francesco Muntoni

Institutions: Columbia University Irving Medical Center, Istituto Giannina Gaslini, Stanford University, University Children’s Hospital Basel, University of Freiburg, University Medical Center Freiburg, NIHR Great Ormond Street Hospital Biomedical Research Centre, University College London, Agostino Gemelli University Polyclinic, 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: W4367304281

FWCI:

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