The First Orally Deliverable Small Molecule for the Treatment of Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is one 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 ski...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
| Published: |
SAGE Publishing
2020-11-01
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| Series: | Neuroscience Insights |
| Online Access: | https://doi.org/10.1177/2633105520973985 |