uniQure released four-year data from its phase 1/2 trial of AMT-130, a gene therapy for Huntington's Disease. The data showed a 44% slowing in disease progression measured by cUHDRS at year 4, though this result was not statistically significant, possibly due to missing data and survivor bias. However, the total functional capacity (TFC) measure, the primary endpoint for the ongoing phase 3 trial, showed over 60% slowing with statistically significant results. These findings support AMT-130's potential to slow Huntington's Disease progression, with further confirmation expected from the phase 3 trial.
The story covers uniQure's updated 4-year trial data for its Huntington's Disease gene therapy. For readers following health-sector stocks, here is Pluang's market snapshot as of Oct 01, 2026 00:03 WIB: out of 100 priced US health stocks, 52 rose and 48 fell. Notable movers include HIMS at USD 30.59 (+6.59%), TWST at USD 196.47 (+5.56%), and SMMT at USD 17.24 (+5.44%).
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