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Spinal muscular atrophy: From approved therapies to future therapeutic targets for personalized medicine
Spinal muscular atrophy (SMA) is a devastating childhood motor neuron disease that, in the most severe cases and when left untreated, leads to death within the first two years of life. Recent therapeutic advances have given hope to families and patients by compensating for the deficiency in survival...
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| Foilsithe in: | Cell Rep Med |
|---|---|
| Main Authors: | , , , |
| Formáid: | Artigo |
| Teanga: | Inglês |
| Foilsithe: |
Elsevier
2021
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| Ábhair: | |
| Rochtain Ar Líne: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8324491/ https://ncbi.nlm.nih.gov/pubmed/34337562 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.xcrm.2021.100346 |
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