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  • October 14, 2026 Frequently asked questions about the PRiSM trial
  • October 14, 2026 Pre-symptomatic arm of divalent siRNA clinical trial now open
  • September 2, 2026 Our public comment on FDA's Expedited IND Pilot Program
  • August 10, 2026 Jeff Carroll: searching for the toxic species in Huntington's disease
  • July 13, 2026 Robert Thorne: blood-brain barrier primer
  • June 2, 2026 Why TAPCJD is so important
  • April 22, 2026 Divalent siRNA clinical trial is now recruiting
  • March 17, 2026 ION717 trial re-opens with 3rd dosing regimen
  • February 18, 2026 Celebrating Byron Caughey, 1957-2026
  • October 8, 2025 Uniqure reports that AMT-130 slows Huntington's disease
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