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Development and Translation of Therapies for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder characterised by widespread loss of lower motor neurons from the spinal cord, leading to progressive weakness and muscle atrophy. SMA is largely caused by homozygous loss of the survival motor neuron (SMN) 1 gene, resulti...

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Detalles Bibliográficos
Autores principales: Hannah K. Shorrock, Thomas H. Gillingwater
Formato: Artigo
Lenguaje:Inglês
Publicado: European Medical Journal 2016-07-01
Colección:European Medical Journal Neurology
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Acceso en línea:http://emjreviews.com/wp-content/uploads/Development-and-Translation-of-Therapies-for-Spinal-Muscular-Atrophy.pdf
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