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Article

Therapeutic strategies for spinal muscular atrophy: SMN and beyond

1 Aug 2017

Abstract excerpt

ABSTRACT Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder characterized by loss of motor neurons and muscle atrophy, generally presenting in childhood. SMA is caused by low levels of the survival motor neuron protein (SMN) due to inactivating mutations in the encoding gene SMN1. A second duplicated gene, SMN2, produces very little but sufficient functional protein for survival. Therapeutic...

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PMID: 28768735
DOI: 10.1242/dmm.030148

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