Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients

Cerebrospinal Fluid Parameters in Antisense Oligonucleotide-Treated Adult 5q-Spinal Muscular Atrophy Patients

Approval of nusinersen, an intrathecally administered antisense oligonucleotide, for the treatment of 5q-spinal muscular atrophy (SMA) marked the beginning of a new therapeutic era in neurological diseases. Changes in routine cerebrospinal fluid (CSF) parameters under nusinersen have only recently b...

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Bibliographic Details
Main Authors: Lars Hendrik Müschen, Alma Osmanovic, Camilla Binz, Konstantin F. Jendretzky, Gresa Ranxha, Paul Bronzlik, Omar Abu-Fares, Flavia Wiehler, Nora Möhn, Martin W. Hümmert, Stefan Gingele, Friedrich Götz, Martin Stangel, Thomas Skripuletz, Olivia Schreiber-Katz, Susanne Petri
Format: Article
Language:English
Published: MDPI AG 2021-02-01
Series:Brain Sciences
Subjects:
spinal muscular atrophy (SMA)
nusinersen
antisense oligonucleotide (ASO)
cerebrospinal fluid (CSF)
lumbar puncture
Online Access:https://www.mdpi.com/2076-3425/11/3/296

Internet

https://www.mdpi.com/2076-3425/11/3/296

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