Experience of Nusinersen in children with proximal spinal muscular atrophy 5q in Moscow region
Background. Proximal spinal muscular atrophy 5q (5q-SMA) is a severe autosomal recessive neuromuscular disorder characterized by progressive flaccid paralysis and muscular atrophy caused by degeneration of α-motor neurons in the anterior horns of the spinal cord resulted from mutations in the SMN1 g...
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| Format: | Article |
| Language: | Russian |
| Published: |
ABV-press
2022-12-01
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| Series: | Русский журнал детской неврологии |
| Subjects: | |
| Online Access: | https://rjdn.abvpress.ru/jour/article/view/404 |
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