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The Identification of Novel Biomarkers Is Required to Improve Adult SMA Patient Stratification, Diagnosis and Treatment

Spinal muscular atrophy (SMA) is currently classified into five different subtypes, from the most severe (type 0) to the mildest (type 4) depending on age at onset, best motor function achieved, and copy number of the SMN2 gene. The two recent approved treatments for SMA patients revolutionized thei...

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Detalles Bibliográficos
Autores principales: Smeriglio, Piera, Langard, Paul, Querin, Giorgia, Biferi, Maria Grazia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7564782/
https://www.ncbi.nlm.nih.gov/pubmed/32751151
http://dx.doi.org/10.3390/jpm10030075