Clinicogenetic characterization and response to disease-modifying therapies in spinal muscular atrophy: real-world experience from a reference center in Southern Brazil
Objective: Spinal Muscular Atrophy linked to chromosome 5q (SMA) is an autosomal recessive neurodegenerative disease characterized by progressive proximal muscle atrophy and weakness. This study addresses the scarcity of research on novel disease-modifying therapies for SMA in Latin America by repor...
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Main Authors: | , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2025-01-01
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Series: | Jornal de Pediatria |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S0021755724001268 |
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