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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...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Springer International Publishing
2018
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| 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 |