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Effect of Nusinersen in a late onset spinal muscular atrophy patient for 14 months: A case report
RATIONALE: Spinal muscular atrophy (SMA) is a genetic disorder caused by genetic defect of SMN1 gene. SMA was an untreatable disease until 2016, when Nusinersen an antisense oligonucleotide therapy was approved for treatment. We report the effect of Nusinersen in a late onset SMA for 14 months. PATI...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Lippincott Williams & Wilkins
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7793442/ https://www.ncbi.nlm.nih.gov/pubmed/33429824 http://dx.doi.org/10.1097/MD.0000000000024236 |