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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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Bibliografiske detaljer
Udgivet i:J Pediatr Pharmacol Ther
Main Authors: Neil, Erin E., Bisaccia, Elizabeth K.
Format: Artigo
Sprog:Inglês
Udgivet: Pediatric Pharmacy Advocacy Group 2019
Fag:
Online adgang: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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