JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam
Work
Year: 2024
Type: article
Abstract: Risdiplam is a once-daily oral, survival of motor neuron 2 ( SMN2 ) splicing modifier approved for the treatment of spinal muscular atrophy (SMA). JEWELFISH (NCT03032172) investigated the safety, tol... more
Source: Journal of Neurology
Institutions Columbia University Irving Medical Center, University of Genoa, Istituto Giannina Gaslini, Palo Alto University, Stanford University +11 more
Cites: 23
Cited by: 3
Related to: 10
FWCI: 4.406
Citation percentile (by year/subfield): 80.69
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Sustainable Development Goal Good health and well-being
Open Access status: hybrid
APC paid (est): $4,190
Funder F. Hoffmann-La Roche