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Comment
. 2022 Aug 16;3(8):100725.
doi: 10.1016/j.xcrm.2022.100725.

Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy

Anna A L Motyl  1 , Thomas H Gillingwater  2
Affiliations
Comment

Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy

Anna A L Motyl et al. Cell Rep Med. .

Abstract

Two new studies by Strauss et al. demonstrated safe and effective pre-symptomatic delivery of gene therapy in children with spinal muscular atrophy (SMA).1,2 These results highlight the importance of newborn screening programs and early therapy delivery for SMA.

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Conflict of interest statement

Declaration of interests T.H.G. has provided consultancy and advisory services concerning SMA therapies to Roche and Novartis. T.H.G. is chair of the clinical & scientific advisory board of SMA Europe.

Comment on

References

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    1. 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., et al. Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial. Nat. Med. 2022;28:1390–1397. doi: 10.1038/s41591-022-01867-3. - DOI - PMC - PubMed
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