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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.
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Elsevier
2022
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| 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 |