Longitudinal data collection in pediatric and adult patients with 5q spinal muscular atrophy in Latin America: LATAM RegistrAME study - a clinical registry study protocol

Longitudinal data collection in pediatric and adult patients with 5q spinal muscular atrophy in Latin America: LATAM RegistrAME study - a clinical registry study protocol

ABSTRACT Spinal muscular atrophy is a rare hereditary neurodegenerative disease characterized by progressive motor neuron loss. The most common form of SMA is linked to 5q (5q-SMA) and is classified into subtypes according to the age of onset and maximum motor function achieved. The severity ranges...

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Main Authors: Elice Carneiro Batista, Edmar Zanoteli, Frederico Monfardini, Gustavo Prado dos Santos, Gisele Sampaio Silva, Otávio Berwanger, Luiz Vicente Rizzo, Henrique Andrade Rodrigues da Fonseca
Format: Article
Language:English
Published: Instituto Israelita de Ensino e Pesquisa Albert Einstein 2024-12-01
Series:Einstein (São Paulo)
Subjects:
Muscular atrophy, spine
5q-SMA
Rare disease
Motor milestone
Online Access:http://www.scielo.br/scielo.php?script=sci_arttext&pid=S1679-45082024000100902&lng=en&tlng=en
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