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Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs
Spinal muscular atrophy (SMA) is a rare, inherited neuromuscular disease caused by deletion and/or mutation of the Survival of Motor Neuron 1 (SMN1) gene. A second gene, SMN2, produces low levels of functional SMN protein that are insufficient to fully compensate for the lack of SMN1. Risdiplam (RG7...
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| Publicado no: | Pharmacol Res Perspect |
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| Main Authors: | , , , , , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
John Wiley and Sons Inc.
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6262736/ https://ncbi.nlm.nih.gov/pubmed/30519476 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/prp2.447 |
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