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Prognostic Factors and Treatment‐Effect Modifiers in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease characterized by loss of motor neurons and muscle atrophy. Untreated infants with type 1 SMA do not achieve major motor milestones, and death from respiratory failure typically occurs before 2 years of age. Individuals with t...

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Detalles Bibliográficos
Autores principales:	Baranello, Giovanni, Gorni, Ksenija, Daigl, Monica, Kotzeva, Anna, Evans, Rachel, Hawkins, Neil, Scott, David A., Mahajan, Anadi, Muntoni, Francesco, Servais, Laurent
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Reviews
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9292571/ https://www.ncbi.nlm.nih.gov/pubmed/33792051 http://dx.doi.org/10.1002/cpt.2247

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