STAT+: Why a Huntington’s patient isn’t fazed by the waning effect of UniQure’s gene therapy
Questions about the durability of an experimental Huntington's disease therapy matter to Wall Street. They matter less to those directly affected by the disease.
Lauren Holder, an advocate for the Huntington's disease community, shared her thoughts on UniQure's gene therapy clinical update. The treatment, known as AMT-130, demonstrated a 44% reduction in disease progression over a four-year period in 12 treated patients. However, the effect of AMT-130 appeared to diminish from the third to fourth year, leading to some questions about the therapy's long-term durability. While this decline in treatment efficacy may be concerning, UniQure has not expressed alarm over the issue.
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