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Advances in therapeutic development for spinal muscular atrophy
Spinal muscular atrophy (SMA) is a leading genetic cause of infant mortality. The disease originates from low levels of SMN protein due to deletion and/or mutations of SMN1 coupled with the inability of SMN2 to compensate for the loss of SMN1. While SMN1 and SMN2 are nearly identical, SMN2 predomina...
Sparad:
| I publikationen: | Future Med Chem |
|---|---|
| Huvudupphovsmän: | , , |
| Materialtyp: | Artigo |
| Språk: | Inglês |
| Publicerad: |
2014
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| Ämnen: | |
| Länkar: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4356243/ https://ncbi.nlm.nih.gov/pubmed/25068989 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4155/fmc.14.63 |
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