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ISS-N1 makes the First FDA-approved Drug for Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is one of the leading genetic diseases of children and infants. SMA is caused by deletions or mutations of Survival Motor Neuron 1 (SMN1) gene. SMN2, a nearly identical copy of SMN1, cannot compensate for the loss of SMN1 due to predominant skipping of exon 7. While var...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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De Gruyter Open
2017
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5382937/ https://www.ncbi.nlm.nih.gov/pubmed/28400976 http://dx.doi.org/10.1515/tnsci-2017-0001 |