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One-time gene therapy for Huntington's disease.

1 report so far. New developments are added to this thread as they happen.

  1. Sep 2, 2026
    APPROVAL REQUESTED IN THE US AND UKApproval filing · US and UK

    One-time brain gene therapy for Huntington's is sent for US, UK approval

    uniQure has asked US and UK regulators to approve its one-time Huntington's gene therapy. The request rests on small early trials compared with untreated patients from outside the studies.