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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...

詳細記述

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書誌詳細
主要な著者: Lorson, Monique A, Lorson, Christian L
フォーマット: Artigo
言語:Inglês
出版事項: 2012
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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