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., Tauscher-Wisniewski, S., McGill, B. E., & Macek, T. A. (2022). Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial. Nature Medicine, 28(7), 1381–1389. https://doi.org/10.1038/s41591-022-01866-4
Subjects:
Gene Therapy for Spinal Muscular Atrophy
(OpenAlex Topic)
RNA Methylation and Modification in Gene Expression
(OpenAlex Topic)
Etiology and Management of Abdominal Wall Defects
(OpenAlex Topic)
Muscular Atrophy, Spinal
(MeSH)
Publication Type:
Article
Unique ID:
10.1038/s41591-022-01866-4
PMID:
Journal:
Publication Date:
Data Source:
Scopus