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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| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Romanian Association of Balneology, Editura Balneara
2025-12-01
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| Series: | Balneo and PRM Research Journal |
| Subjects: | |
| Online Access: | http://bioclima.ro/Balneo754.pdf |
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