Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Strauss, K. A., Farrar, M. A., Muntoni, F., Saito, K., Mendell, J. R., Servais, L., McMillan, H. J., Finkel, R. S., Swoboda, K. J., Kwon, J. M., Zaidman, C. M., Chiriboga, C. A., Iannaccone, S. T., Krueger, J. M., Parsons, J. A., Shieh, P. B., Kavanagh, S., Wigderson, M., Tauscher-Wisniewski, S., … Macek, T. A. (2022). Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. Nature Medicine, 28(7), 1390–1397. https://doi.org/10.1038/s41591-022-01867-3
Authors:
Kevin A. Strauss
Michelle A. Farrar
Francesco Muntoni
Kayoko Saito
Jerry R. Mendell
Laurent Servais
Hugh J. McMillan
Richard S. Finkel
Kathryn J. Swoboda
Jennifer M. Kwon
Craig M. Zaidman
Claudia A. Chiriboga
Susan T. Iannaccone
Jena M. Krueger
Julie A. Parsons
Perry B. Shieh
Sarah Kavanagh
Melissa Wigderson
Sitra Tauscher-Wisniewski
Bryan E. McGill
Thomas A. Macek
Affiliated Authors:
Claudia A. Chiriboga
Publication Type:
Article
Unique ID:
10.1038/s41591-022-01867-3
PMID:
Journal:
Publication Date:
Data Source:
Scopus

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