Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive review

Abstract Background: Adult-onset spinal muscular atrophy (SMA) represents an expanding group of inherited neurodegenerative disorders in clinical practice. Objective: This review aims to synthesize the main clinical, genetic, radiological, biochemical, and neurophysiological aspects related to the...

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Main Authors: Wladimir Bocca Vieira de Rezende Pinto, Paulo Victor Sgobbi de Souza, Bruno Mattos Lombardi Badia, Igor Braga Farias, José Marcos Vieira de Albuquerque Filho, Eduardo Augusto Gonçalves, Roberta Ismael Lacerda Machado, Acary Souza Bulle Oliveira
Format: Article
Language:English
Published: Thieme Revinter Publicações 2021-01-01
Series:Arquivos de Neuro-Psiquiatria
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Online Access:http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2021001000912&tlng=en
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author Wladimir Bocca Vieira de Rezende Pinto
Paulo Victor Sgobbi de Souza
Bruno Mattos Lombardi Badia
Igor Braga Farias
José Marcos Vieira de Albuquerque Filho
Eduardo Augusto Gonçalves
Roberta Ismael Lacerda Machado
Acary Souza Bulle Oliveira
author_facet Wladimir Bocca Vieira de Rezende Pinto
Paulo Victor Sgobbi de Souza
Bruno Mattos Lombardi Badia
Igor Braga Farias
José Marcos Vieira de Albuquerque Filho
Eduardo Augusto Gonçalves
Roberta Ismael Lacerda Machado
Acary Souza Bulle Oliveira
author_sort Wladimir Bocca Vieira de Rezende Pinto
collection DOAJ
description Abstract Background: Adult-onset spinal muscular atrophy (SMA) represents an expanding group of inherited neurodegenerative disorders in clinical practice. Objective: This review aims to synthesize the main clinical, genetic, radiological, biochemical, and neurophysiological aspects related to the classical and recently described forms of proximal SMA. Methods: The authors performed a non-systematic critical review summarizing adult-onset proximal SMA presentations. Results: Previously limited to cases of SMN1-related SMA type 4 (adult form), this group has now more than 15 different clinical conditions that have in common the symmetrical and progressive compromise of lower motor neurons starting in adulthood or elderly stage. New clinical and genetic subtypes of adult-onset proximal SMA have been recognized and are currently target of wide neuroradiological, pathological, and genetic studies. Conclusions: This new complex group of rare disorders typically present with lower motor neuron disease in association with other neurological or systemic signs of impairment, which are relatively specific and typical for each genetic subtype.
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spelling doaj-art-083be35c8d644fd6bbe176abec3f7d822025-08-20T02:01:35ZengThieme Revinter PublicaçõesArquivos de Neuro-Psiquiatria1678-42272021-01-01791091292310.1590/0004-282x-anp-2020-0429Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive reviewWladimir Bocca Vieira de Rezende Pintohttps://orcid.org/0000-0002-0150-525XPaulo Victor Sgobbi de Souzahttps://orcid.org/0000-0002-7416-7108Bruno Mattos Lombardi Badiahttps://orcid.org/0000-0001-7360-0494Igor Braga Fariashttps://orcid.org/0000-0003-2187-6573José Marcos Vieira de Albuquerque Filhohttps://orcid.org/0000-0002-4533-2637Eduardo Augusto Gonçalveshttps://orcid.org/0000-0002-9893-2468Roberta Ismael Lacerda Machadohttps://orcid.org/0000-0002-5842-0062Acary Souza Bulle Oliveirahttps://orcid.org/0000-0002-6986-4937Abstract Background: Adult-onset spinal muscular atrophy (SMA) represents an expanding group of inherited neurodegenerative disorders in clinical practice. Objective: This review aims to synthesize the main clinical, genetic, radiological, biochemical, and neurophysiological aspects related to the classical and recently described forms of proximal SMA. Methods: The authors performed a non-systematic critical review summarizing adult-onset proximal SMA presentations. Results: Previously limited to cases of SMN1-related SMA type 4 (adult form), this group has now more than 15 different clinical conditions that have in common the symmetrical and progressive compromise of lower motor neurons starting in adulthood or elderly stage. New clinical and genetic subtypes of adult-onset proximal SMA have been recognized and are currently target of wide neuroradiological, pathological, and genetic studies. Conclusions: This new complex group of rare disorders typically present with lower motor neuron disease in association with other neurological or systemic signs of impairment, which are relatively specific and typical for each genetic subtype.http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2021001000912&tlng=enMuscular Atrophy, SpinalNeurodegenerative DiseasesMotor Neuron DiseaseNeuromuscular Diseases
spellingShingle Wladimir Bocca Vieira de Rezende Pinto
Paulo Victor Sgobbi de Souza
Bruno Mattos Lombardi Badia
Igor Braga Farias
José Marcos Vieira de Albuquerque Filho
Eduardo Augusto Gonçalves
Roberta Ismael Lacerda Machado
Acary Souza Bulle Oliveira
Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive review
Arquivos de Neuro-Psiquiatria
Muscular Atrophy, Spinal
Neurodegenerative Diseases
Motor Neuron Disease
Neuromuscular Diseases
title Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive review
title_full Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive review
title_fullStr Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive review
title_full_unstemmed Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive review
title_short Adult-onset non-5q proximal spinal muscular atrophy: a comprehensive review
title_sort adult onset non 5q proximal spinal muscular atrophy a comprehensive review
topic Muscular Atrophy, Spinal
Neurodegenerative Diseases
Motor Neuron Disease
Neuromuscular Diseases
url http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0004-282X2021001000912&tlng=en
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