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Advances in therapeutic development for spinal muscular atrophy

Spinal muscular atrophy (SMA) is a leading genetic cause of infant mortality. The disease originates from low levels of SMN protein due to deletion and/or mutations of SMN1 coupled with the inability of SMN2 to compensate for the loss of SMN1. While SMN1 and SMN2 are nearly identical, SMN2 predomina...

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Foilsithe in:Future Med Chem
Main Authors: Howell, Matthew D, Singh, Natalia N, Singh, Ravindra N
Formáid: Artigo
Teanga:Inglês
Foilsithe: 2014
Ábhair:
Rochtain Ar Líne:https://ncbi.nlm.nih.gov/pmc/articles/PMC4356243/
https://ncbi.nlm.nih.gov/pubmed/25068989
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4155/fmc.14.63
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