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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...
Gespeichert in:
| Veröffentlicht in: | J Pediatr Pharmacol Ther |
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| Hauptverfasser: | , |
| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Pediatric Pharmacy Advocacy Group
2019
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| Schlagworte: | |
| Online Zugang: | 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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