Therapeutic advances in 5q-linked spinal muscular atrophy.
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DOI: 10.1590/0004-282x20180011
Abstract: Spinal muscular atrophy (SMA) is a severe and clinically-heterogeneous motor neuron disease caused, in most cases, by a homozygous mutation in the SMN1 gene. Regarding the age of onset and motor involvement, at least four… read more here.
Keywords: advances linked; therapeutic advances; spinal muscular; linked spinal ... See more keywords