2021
DOI: 10.21203/rs.3.rs-564501/v1
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Increased Chitotriosidase1 Concentration Following Nusinersen Treatment in Spinal Muscular Atrophy

Abstract: BackgroundStudies 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 a marginal but significant increase of the serum / CSF albumin ratio (Qalb) with rare cases of communicating hydrocephalus during nusinersen treatment were reported, which confirms the unmet need of an inflammatory biomarker… Show more

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