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Article

Overview of Current Drugs and Molecules in Development for Spinal Muscular Atrophy Therapy

29 Jan 2018

Abstract excerpt

Spinal muscular atrophy (SMA) is a neurodegenerative disease primarily characterized by a loss of spinal motor neurons, leading to progressive paralysis and premature death in the most severe cases. SMA is caused by homozygous deletion of the survival motor neuron 1 (SMN1) gene, leading to low levels of SMN protein. However, a second SMN gene (SMN2) exists, which can be therapeutically targeted to increase SMN...

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PMID: 29380287
DOI: 10.1007/s40265-018-0868-8

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