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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...
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| Pubblicato in: | Neurosci Insights |
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| Autori principali: | , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
SAGE Publications
2020
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7691903/ https://ncbi.nlm.nih.gov/pubmed/33283185 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/2633105520973985 |
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