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Safety, tolerability, and efficacy of PBT2 in Huntington's disease: A phase 2, randomised, double-blind, placebo-controlled trial

Kumar, Rajeev; Ames, D


Background PBT2 is a metal protein-attenuating compound that might reduce metal-induced aggregation of mutant huntingtin and has prolonged survival in a mouse model of Huntington’s disease. We aimed to assess the safety, tolerability, and effi cacy of PBT2 in patients with Huntington’s disease. Methods In this 26-week, randomised, double-blind, placebo-controlled trial, adults (≥25 years old) with early-stage to mid-stage Huntington’s disease were randomly assigned (1:1:1) by a centralised...[Show more]

CollectionsANU Research Publications
Date published: 2015
Type: Journal article
Source: The Lancet Neurology
DOI: 10.1016/S1474-4422(14)70262-5


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