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Article

Spinal muscular atrophy: from tissue specificity to therapeutic strategies

5 Jan 2015

Abstract excerpt

Spinal muscular atrophy (SMA) is the most frequent genetic cause of death in infants and toddlers. All cases of spinal muscular atrophy result from reductions in levels of the survival motor neuron (SMN) protein, and so SMN upregulation is a focus of many preclinical and clinical studies. We examine four issues that may be important in planning for therapeutic success. First, neuromuscular phenotypes in the SMNΔ7...

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PMID: 25705387
DOI: 10.12703/p7-04

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