Posted on: 6 March 2026, source: CNBC UniQure needs to run another study to prove that its gene therapy “actually helps people with Huntington’s disease,” a senior U.S. Food and Drug Administration official said on a call with reporters Thursday. The official, who requested anonymity before discussing sensitive information, confirmed the agency has asked the company to run a placebo controlled trial of its treatment, which is administered directly into the brain. UniQure has said that type of study isn’t ethical because it would require putting people under general anesthesia for hours, a characterization the official disputed. {fastsocialshare}
FDA official calls UniQure’s gene therapy a ‘failed’ treatment for Huntington’s disease
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