Interim report on the safety and efficacy of longer-term treatment with nusinersen in later-onset spinal muscular atrophy (SMA): Results from the shine study
Year: 2019
Type: article
Authors: J. Kirschner, B.T. Darras, M.A. Farrar, E. Mercuri, C.A. Chiriboga, N.L. Kuntz, P.B. Shieh, M. Tulinius, J. Montes, S.P. Reyna, G. Gambino, R. Foster, I. Bhan, J. Wong, W. Farwell
Institutions: University Hospital Bonn, Boston Children's Hospital, UNSW Sydney, Sydney Children's Hospital, Sydney Children’s Hospitals Network, Università Cattolica del Sacro Cuore, Columbia University Irving Medical Center, Lurie Children's Hospital, University of California, Los Angeles, University of Gothenburg, Biogen (United States), Biogen (United Kingdom)
Language: English
Subfield: Genetics
Field: Medicine
Domain: Health Sciences
Open Access status: closed
OpenAlex ID: W2996811033