FDA Under Pressure to Approve UniQure's Huntington's Treatment Amid Advocacy Groups' Plea

Patient advocates for Huntington's disease are urging the FDA to approve UniQure's gene therapy, citing a clinical trial that showed patients progressed 75% slower than a comparison group. The FDA had previously agreed that the trial's results were sufficient for approval but has since changed its stance. The outcome will provide insight into the FDA's flexibility with new treatments for rare diseases.