Study of nusinersen metabolites in the cerebrospinal fluid of children with spinal muscular atrophy using ultra-high-performance liquid chromatography coupled with quadrupole-time-of-flight mass spectrometry

Author:

Studzińska Sylwia12ORCID,Błachowicz Oliwia1,Bocian Szymon1ORCID,Kalisz Oktawia1ORCID,Jaworska Aleksandra1,Szymarek Jakub3,Mazurkiewicz-Bełdzińska Maria3

Affiliation:

1. Chair of Environmental Chemistry and Bioanalytics, Faculty of Chemistry, Nicolaus Copernicus University in Toruń, 7 Gagarin St., 87-100 Toruń, Poland

2. Centre for Modern Interdisciplinary Technologies, Nicolaus Copernicus University in Toruń, 4 Wilenska St., 87-100 Toruń, Poland

3. Department of Developmental Neurology, Medical University of Gdansk, 7 Dębinki Str., PL-80-952, Gdańsk, Poland

Abstract

This study aimed to analyze nusinersen metabolites in cerebrospinal fluid samples using ion-pair reversed-phase ultrahigh-performance liquid chromatography coupled with quadrupole time-of-flight mass spectrometry.

Publisher

Royal Society of Chemistry (RSC)

Reference22 articles.

1. Nusinersen: First Global Approval

2. US Food and Drug Administration , 2016 , 2016 Novel Drugs Summary. https://Www.Fda.Gov/News-Events/Press-Announcements/Fda-Approves-First-Drug-Spinal-Muscular-Atrophy

3. European Medicines Agency , 2017 , Spinraza. https://www.Ema.Europa.Eu/En/Homepage

4. Nusinersen Induces Disease-Severity-Specific Neurometabolic Effects in Spinal Muscular Atrophy

5. Molecular Biomarkers for the Diagnosis, Prognosis, and Pharmacodynamics of Spinal Muscular Atrophy

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