Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease.
Movement disorders : official journal of the Movement Disorder Society
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Stoker, T. B., Andresen, K., & Barker, R. (2021). Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease.. Movement disorders : official journal of the Movement Disorder Society, 36 (1), 263-264. https://doi.org/10.1002/mds.28359
Huntington’s disease (HD) is a genetic disorder caused by an expanded CAG repeat in the huntingtin gene, and although there are currently no disease-modifying treatments, there is much excitement about the prospect of treatments targeting huntingtin expression. In a phase I/2A trial of an antisense oligonucleotide (ASO) treatment (Tominersen), no serious adverse events were recorded, and there was a dose-dependent reduction in cerebrospinal fluid (CSF) huntingtin levels1. In an open-label extension (OLE) study, patients received monthly or bimonthly Tominersen, with preliminary data confirming the reduction in mutant huntingtin levels2. Here we report on a unique major adverse effect occurring during this OLE.
Humans, Huntington Disease, Hydrocephalus, Oligonucleotides, Antisense, Huntingtin Protein
Funding sources and conflict of interest – This trial was funded initially by Ionis and subsequently by Roche. The authors received no additional funding for this work and the authors declare that there are no conflicts of interest relevant to this work. Financial disclosures - RAB is supported by the National Institute for Health Research (NIHR) Cambridge Biomedical Research Centre - 146281 (the views expressed are those of the author(s) and not necessarily those of the NIHR or the Department of Health and Social Care) and MRC/WT Stem Cell Institute (203151/Z/16/Z).
Cambridge University Hospitals NHS Foundation Trust (CUH) (146281)
Wellcome Trust (203151/Z/16/Z)
Medical Research Council (MC_PC_17230)
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External DOI: https://doi.org/10.1002/mds.28359
This record's URL: https://www.repository.cam.ac.uk/handle/1810/311035
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