UniQure’s Huntington’s disease gene therapy AMT-130 came under fresh scrutiny after additional four-year follow-up data suggested the magnitude of benefit may have waned relative to earlier analyses. According to UniQure’s update, high-dose treatment slowed disease progression by 44% versus a matched external control group drawn from a large natural history dataset, but the company reported that the difference was not statistically significant. Analysts and investors focused on whether the durability profile could complicate the therapy’s ongoing regulatory review. The renewed questions land as the FDA prepares to review UniQure’s marketing application, putting pressure on whether functional outcomes and secondary endpoints remain persuasive. The update triggered market reaction alongside broader investor sensitivity to long-term neurodegeneration endpoints in gene therapy programs.