The US Food and Drug Administration told uniQure it requires a prospective, randomized, double‑blind, sham surgery‑controlled study to support approval of AMT‑130, the AAV‑based Huntington’s disease gene therapy. The agency concluded that Phase I/II data compared to external controls are insufficient for primary effectiveness evidence, prompting calls from clinicians and patient advocates who argue a long sham‑controlled trial could leave some participants untreated for years. Edward Wild of UCL highlighted the ethical tension between rigorous evidence standards and the therapy’s life‑prolonging effects reported in early trials.