Spinal Muscular Atrophy Treatment in Patients Identified by Newborn Screening—A Systematic Review

Background: In spinal muscular atrophy, clinical trial results indicated that disease-modifying treatments are highly effective when given prior to symptom onset, which has prompted newborn screening programs in growing number of countries. However, prognosis of those patients cannot be inferred fro...

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Detalles Bibliográficos
Autores principales: Aragon-Gawinska, Karolina, Mouraux, Charlotte, Dangouloff, Tamara, Servais, Laurent
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Systematic Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10379202/
https://www.ncbi.nlm.nih.gov/pubmed/37510282
http://dx.doi.org/10.3390/genes14071377

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