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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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Bibliografische gegevens
Hoofdauteurs: Lorson, Monique A, Lorson, Christian L
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2012
Onderwerpen:
Online toegang: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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