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

Anna A L Motyl; Thomas H Gillingwater

doi:10.1016/j.xcrm.2022.100725

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

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

Authors

Anna A L Motyl¹, Thomas H Gillingwater²

Affiliations

¹ Edinburgh Medical School: Biomedical Sciences, University of Edinburgh, Edinburgh, UK; Euan MacDonald Centre for Motor Neuron Disease, University of Edinburgh, Edinburgh, UK.
² Edinburgh Medical School: Biomedical Sciences, University of Edinburgh, Edinburgh, UK; Euan MacDonald Centre for Motor Neuron Disease, University of Edinburgh, Edinburgh, UK. Electronic address: t.gillingwater@ed.ac.uk.

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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MeSH terms

Child
Humans
Infant, Newborn
Muscular Atrophy, Spinal* / diagnosis
Neonatal Screening / methods