Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy

Spinal muscular atrophy (SMA) is a neurodegenerative disease primarily characterized by a loss of spinal motor neurons, leading to progressive paralysis and premature death in the most severe cases. SMA is caused by homozygous deletion of the survival motor neuron 1 (SMN1) gene, leading to low level...

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Detalles Bibliográficos
Autores principales: Shorrock, Hannah K., Gillingwater, Thomas H., Groen, Ewout J. N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2018
Materias:
Leading Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5829132/
https://www.ncbi.nlm.nih.gov/pubmed/29380287
http://dx.doi.org/10.1007/s40265-018-0868-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5829132/
https://www.ncbi.nlm.nih.gov/pubmed/29380287
http://dx.doi.org/10.1007/s40265-018-0868-8

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