Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy.
editorial · Level V
Where this comes from
- Record sourced from PubMed, PMID 35977471.
- Also identified by DOI 10.1016/j.xcrm.2022.100725 and PMC identifier 9418840.
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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).<sup>1</sup><sup>,</sup><sup>2</sup> These results highlight the importance of newborn screening programs and early therapy delivery for SMA.
Medical subject headings
- Muscular Atrophy, Spinal