Efficacy of Nusinersen Treatment in Type 1, 2, and 3 Spinal Muscular Atrophy: Real-World Data from a Single-Center Study
Background: Spinal muscular atrophy (SMA) is an inherited neuromuscular disease characterized by progressive muscle weakness and atrophy due to the absence of the survival motor neuron 1 (<i>SMN1</i>) gene. SMA is classified into types 0 through 4 based on the age of symptom onset and th...
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| Main Authors: | , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2024-10-01
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| Series: | Neurology International |
| Subjects: | |
| Online Access: | https://www.mdpi.com/2035-8377/16/6/96 |
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