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Nusinersen: A Novel Antisense Oligonucleotide for the Treatment of Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) encompasses a group of autosomal recessively inherited degenerative neuromuscular disorders. They range in severity from neonatal onset with rapidly progressive weakness and early mortality (SMA-1), to onset in infancy (SMA-2), to adolescent/adult onset with indolent cl...
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| Publicado no: | J Pediatr Pharmacol Ther |
|---|---|
| Main Authors: | , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Pediatric Pharmacy Advocacy Group
2019
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6510522/ https://ncbi.nlm.nih.gov/pubmed/31093018 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.5863/1551-6776-24.3.194 |
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