Predictive Clinical-Biological Markers Over the First 3 Years of Nusinersen Treatment in SMA Type 1 Patients

Predictive Clinical-Biological Markers Over the First 3 Years of Nusinersen Treatment in SMA Type 1 Patients

Werdnig-Hoffmann disease, or type 1 Spinal Muscular Atrophy (SMA), is caused by insufficient SMN protein synthesis due to a genetic defect. Symptoms appear within the first 6 months of life, and without ventilatory support, life expectancy averages 2 years. This study aimed to monitor pNF-H neurofil...

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Bibliographic Details
Main Authors: Mihaela Badina, Andrada Mirea, Corina Sporea, Gabriel Cristian Bejan
Format: Article
Language:English
Published: Romanian Association of Balneology, Editura Balneara 2025-12-01
Series:Balneo and PRM Research Journal
Subjects:
spinal muscular atrophy
spinraza nusinersen
pnf-h neurofilaments
cere-brospinal fluid
creatinine
motor evolution
predictive markers
neurodegenerative disease
genetic mutation smn
Online Access:http://bioclima.ro/Balneo754.pdf
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http://bioclima.ro/Balneo754.pdf

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