Related Experiment Video
Updated: May 27, 2025

Efficient and Scalable Production of Full-length Human Huntingtin Variants in Mammalian Cells using a Transient Expression System
Published on: December 10, 2021
Huntington's Disease Clinical Trials Update: September 2024
Mena Farag1, Sarah J Tabrizi1, Edward J Wild1
1Huntington's Disease Centre, UCL Queen Square Institute of Neurology, London, UK.
Abstract:
In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing extension study of PTC518 from PTC Therapeutics, including 12-month interim results from the parent study. We also discuss 24-month interim results from uniQure's AMT-130 program and 28-week follow-up results from Wave Life Sciences' SELECT-HD clinical trial of WVE-003. Additionally, we provide a comprehensive listing of all currently registered and ongoing clinical trials in Huntington's disease.
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