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Huntington's Disease

FDA Agrees Three‑Year Phase I/II Data Can Support uniQure’s AMT‑130 Accelerated BLA

It opens a route for accelerated approval with negotiations underway over the design of a required post‑approval confirmatory trial.

FDA Meeting Minutes Undercut uniQure’s Plan to File Huntington’s Gene Therapy for Approval

Gene Therapy Slows Huntington’s Progression by 75% in Early Trial, uniQure Says