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New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges

Spinal muscular atrophy (SMA) is one of the most common autosomal recessive diseases with progressive weakness of skeletal and respiratory muscles, leading to significant disability. The disorder is caused by mutations in the survival motor neuron 1 (SMN1) gene and a consequent decrease in the SMN p...

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Detalles Bibliográficos
Autores principales: Messina, Sonia, Sframeli, Maria
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7408870/
https://www.ncbi.nlm.nih.gov/pubmed/32668756
http://dx.doi.org/10.3390/jcm9072222

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