Therapeutic advances in 5q-linked spinal muscular atrophy

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 distinct clinical phenotypes have been recognized. This clinical vari...

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Bibliographic Details
Main Authors: Umbertina Conti Reed, Edmar Zanoteli
Format: Article
Language:English
Published: Thieme Revinter Publicações
Series:Arquivos de Neuro-Psiquiatria
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Online Access:http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2018000400265&lng=en&tlng=en
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