A multidisciplinary approach to dosing nusinersen for spinal muscular atrophy

BACKGROUND: In December 2016, nusinersen gained FDA approval as the first pharmacologic treatment for spinal muscular atrophy (SMA), a disorder of motor neurons and the leading genetic cause of infant mortality. Nusinersen's intrathecal delivery requirement, strict dosage protocol, and accelera...

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Bibliografiske detaljer
Udgivet i:Neurol Clin Pract
Main Authors: Zingariello, Carla D., Brandsema, John, Drum, Elizabeth, Henderson, Alicia A., Dubow, Scott, Glanzman, Allan M., Mayer, Oscar, Yum, Sabrina W., Kichula, Elizabeth A.
Format: Artigo
Sprog:Inglês
Udgivet: Lippincott Williams & Wilkins 2019
Fag:
Research
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6814423/
https://ncbi.nlm.nih.gov/pubmed/31750028
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/CPJ.0000000000000718
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