Type-1 spinal muscular atrophy cohort before and after disease-modifying therapies

Type-1 spinal muscular atrophy cohort before and after disease-modifying therapies

Background Spinal muscular atrophy (SMA-5q) is a neurodegenerative disease characterized by progressive muscle atrophy, hypotonia, and weakness, with SMA 1 presenting symptoms within the first 6 months of life. Disease-modifying therapies have been approved, with better outcomes with earl...

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Bibliographic Details
Main Authors: Brenda Klemm Arci Mattos de Freitas Alves, Alexandra Prufer de Queiroz Campos Araujo, Flávia Nardes dos Santos, Márcia Gonçalves Ribeiro
Format: Article
Language:English
Published: Thieme Revinter Publicações 2024-11-01
Series:Arquivos de Neuro-Psiquiatria
Subjects:
Survival of Motor Neuron 1 Protein
Muscular Atrophy, Spinal
Genetic Therapy
Proteína 1 de Sobrevivência do Neurônio Motor
Atrofia Muscular Espinhal
Terapia Genética
Online Access:http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1791757
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http://www.thieme-connect.de/DOI/DOI?10.1055/s-0044-1791757

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