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Why a Huntington's Patient Isn't Fazed by UniQure's Waning Gene Therapy
Key Takeaways
- STAT News reports that a Huntington's patient is undeterred by signs that UniQure's gene therapy benefit may wane over time.
- Durability questions weigh heavily on Wall Street because a fading effect undermines the one-time-treatment economic model.
- Patients measure benefit against the trajectory of an untreatable, progressive disease rather than against a decade of durable response.
- The durability debate extends across gene therapy programs, affecting regulators, payers, re-dosing risk and trial design.
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DT Editorial Team··via statnews.com
