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

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.

Detalles Bibliográficos
Autores principales: Motyl, Anna A.L., Gillingwater, Thomas H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Spotlight
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9418840/
https://www.ncbi.nlm.nih.gov/pubmed/35977471
http://dx.doi.org/10.1016/j.xcrm.2022.100725
Descripción
Sumario: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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