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SMN-inducing compounds for the treatment of spinal muscular atrophy
Spinal muscular atrophy (SMA) is a leading genetic cause of infant mortality. A neurodegenerative disease, it is caused by loss of SMN1, although low, but essential, levels of SMN protein are produced by the nearly identical gene SMN2. While no effective treatment or therapy currently exists, a new...
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| 主要な著者: | , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2012
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3589915/ https://ncbi.nlm.nih.gov/pubmed/23157239 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4155/fmc.12.131 |
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