Hydrocephalus Complicating Intrathecal Antisense Oligonucleotide Therapy for Huntington's Disease

Abstract excerpt

Huntington's disease 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...

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