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Small Molecules in Development for the Treatment of Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease resulting from pathologically low levels of survival motor neuron (SMN) protein. The majority of mRNA from the SMN2 allele undergoes alternative splicing and excludes critical codons, causing an SMN protein deficiency....
Guardat en:
| Publicat a: | J Med Chem |
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| Autors principals: | , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
2016
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5744254/ https://ncbi.nlm.nih.gov/pubmed/27490705 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/acs.jmedchem.6b00670 |
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