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The Japan Registry for Adult Subjects of Spinal Muscular Atrophy (jREACT-SMA): Protocol for a Longitudinal Observational Study

BACKGROUND: Spinal muscular atrophy (SMA) is an autosomal recessive genetic neuromuscular disorder with progressive muscle weakness and atrophy, mainly caused by lower motor neuron degeneration resulting from decreased levels of the survival motor neuron protein. Recently, 3 disease-modifying therap...

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Detalles Bibliográficos
Autores principales:	Sahashi, Kentaro, Hashizume, Atsushi, Kuwatsuka, Yachiyo, Chinen, Madoka, Saotome-Nakamura, Ai, Ando, Masahiko, Katsuno, Masahisa
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	JMIR Publications 2022
Materias:	Protocol
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9801261/ https://www.ncbi.nlm.nih.gov/pubmed/36520510 http://dx.doi.org/10.2196/38878

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