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Comment
. 2022 Jul;28(7):1348-1349.
doi: 10.1038/s41591-022-01889-x.

Early treatment is a lifeline for infants with SMA

Charlotte J Sumner  1   2 , Thomas O Crawford  3   4
Affiliations
Comment

Early treatment is a lifeline for infants with SMA

Charlotte J Sumner et al. Nat Med. 2022 Jul.

Abstract

In the Phase 3 SPRINT trial, pre-symptomatic gene therapy demonstrated impressive clinical outcomes in infants with a genetic diagnosis of SMA; long-term safety follow-up of these patients must now be a key priority.

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Conflict of interest statement

Author conflict of interests: CJS has been a consultant to Novartis, Ionis Pharmaceuticals, Biogen, PTC Therapeutics, Roche, Genentech, Cytokinetics, Sarepta, Nura Bio, Atalanta, Shift, Argenx, Biomarin, Scholar Rock, GenEdit, Epirium, and Capsigen. CJS has received research grant from Ionis Pharmaceuticals and currently receives grant support from Roche and Biogen. CJS is a coholder of 2 pending patent applications (BIOL0274USA and BIOL0293WO) with Ionis Pharmaceuticals on antisense oligonucleotides targeting SMN-AS1. CJS receives royalties from Elsevier for the book Spinal Muscular Atrophy: Disease Mechanisms and Therapy (editors, CJ Sumner, S Paushkin, CP Ko; Elsevier, 2017). TOC has been a consultant to Avexis/Novartis, Biogen, Catalyst, Cytokinetics, Erydel, Genentech, Ionis, and Scholar Rock. He is/has been a site principal or co–principal investigator for the Biogen EMBRACE, NURTURE, and DEVOTE clinical trials, the Avexis/Novartis STR1VE and STRONG clinical trials, and individual clinical trials with Catabasis, Catalyst, Cytokinetics, Santherra, and Sarepta.

Figures

Figure 1.
Figure 1.. Onasemnogene abeparvovec (OA)
an AAV9 SMN gene replacement therapy, is administered as a one-time intravenous injection. Transduction of motor neurons likely results in improved motor neuron development, function, and survival with the potential for near normal attainment of motor milestones. Whether OA remains effective and safe throughout life requires further assessment.
See this image and copyright information in PMC

Comment on

References

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