Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy

Abstract Background Studies regarding the impact of (neuro)inflammation and inflammatory response following repetitive, intrathecally administered antisense oligonucleotides (ASO) in 5q-associated spinal muscular atrophy (SMA) are sparse. Increased risk of hydrocephalus in untreated SMA patients and...

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Bibliographic Details
Main Authors: Maren Freigang, Petra Steinacker, Claudia Diana Wurster, Olivia Schreiber-Katz, Alma Osmanovic, Susanne Petri, Jan Christoph Koch, Kevin Rostásy, Björn Falkenburger, Albert Christian Ludolph, Markus Otto, Andreas Hermann, René Günther
Format: Article
Language:English
Published: BMC 2021-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
SMA
ASO
Online Access:https://doi.org/10.1186/s13023-021-01961-8