UniQure’s Huntington’s disease gene therapy AMT-130 is facing renewed durability questions after additional four-year follow-up data showed a smaller magnitude of benefit versus earlier analyses. Multiple reports highlighted that while the therapy continued to slow disease progression, the size of the effect declined at the four-year check-in. The updated picture could complicate regulatory discussions as FDA begins review of UniQure’s marketing application, with analysts focusing on whether durability remains sufficiently consistent for approval. Investors reacted negatively, with the stock falling sharply on the new dataset. The case illustrates how long-duration follow-up can reshape both clinical interpretation and regulatory momentum for one-time, brain-targeted gene therapies.