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The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is 1 of the leading causes of infant mortality. SMA is mostly caused by low levels of Survival Motor Neuron (SMN) protein due to deletion of or mutation in the SMN1 gene. Its nearly identical copy, SMN2, fails to compensate for the loss of SMN1 due to predominant skippi...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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SAGE Publications
2020
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7691903/ https://www.ncbi.nlm.nih.gov/pubmed/33283185 http://dx.doi.org/10.1177/2633105520973985 |