Muscle: an independent contributor to the neuromuscular spinal muscular atrophy disease phenotype
Spinal muscular atrophy (SMA) is a pediatric-onset neuromuscular disorder caused by insufficient survival motor neuron (SMN) protein. SMN restorative therapies are now approved for the treatment of SMA; however, they are not curative, likely due to a combination of imperfect treatment timing, inadeq...
| Published in: | JCI Insight |
|---|---|
| Main Authors: | , , , |
| Format: | Article |
| Language: | English |
| Published: |
American Society for Clinical investigation
2023-09-01
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| Online Access: | https://doi.org/10.1172/jci.insight.171878 |