Assistant Professor Rachel Harding in Leslie Dan Faculty of Pharmacy

Reposted from HDBuzz.net

UniQure has announced positive top-line results from its Phase I/II trial of AMT-130, a one-time gene therapy being tested in people with Huntington’s disease (HD). Topline data is a summary of the key results from a study that is released quickly after data becomes available to the company at a specified timepoint. In this update, uniQure reports that symptom progression is being significantly slowed by the drug, and the primary endpoint of the trial was met. This is the first time any drug has been shown to alter the course of HD in people in a clinical trial. HDBuzz caught up with uniQure’s Chief Medical Officer Walid Abi-Saab and CEO Matt Kapusta to get clarity for the HD community around this update. Let’s get into the details of this drug and the update uniQure have shared.

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