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
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author Motyl, Anna A.L.
Gillingwater, Thomas H.
author_facet Motyl, Anna A.L.
Gillingwater, Thomas H.
author_sort Motyl, Anna A.L.
collection PubMed
description 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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spelling pubmed-94188402022-08-28 Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy Motyl, Anna A.L. Gillingwater, Thomas H. Cell Rep Med Spotlight 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. Elsevier 2022-08-16 /pmc/articles/PMC9418840/ /pubmed/35977471 http://dx.doi.org/10.1016/j.xcrm.2022.100725 Text en © 2022. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Spotlight
Motyl, Anna A.L.
Gillingwater, Thomas H.
Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy
title Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy
title_full Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy
title_fullStr Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy
title_full_unstemmed Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy
title_short Timing is everything: Clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy
title_sort timing is everything: clinical evidence supports pre-symptomatic treatment for spinal muscular atrophy
topic Spotlight
url 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
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