Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease
| Autor: | Thomas B Stoker, Katie E.R. Andresen, Roger A. Barker |
|---|---|
| Přispěvatelé: | Stoker, Thomas B [0000-0001-5186-7630], Apollo - University of Cambridge Repository |
| Rok vydání: | 2020 |
| Předmět: |
0301 basic medicine
congenital hereditary and neonatal diseases and abnormalities medicine.medical_specialty Regular Issue Articles Intrathecal 03 medical and health sciences 0302 clinical medicine Antisense Oligonucleotide Therapy Huntington's disease mental disorders medicine Humans Huntingtin Protein business.industry Conflict of interest Oligonucleotides Antisense Letters: New Observations medicine.disease nervous system diseases Hydrocephalus Huntington Disease 030104 developmental biology Neurology Research centre Financial Disclosures Family medicine Social care Neurology (clinical) business 030217 neurology & neurosurgery |
| Zdroj: | Movement Disorders |
| ISSN: | 1531-8257 0885-3185 |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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