When we first reported on this cohort in 2020, it was a simple, happy story: https://t.co/1CirVtdWYe CSF PrP had good test-retest stability, meaning it could be a pharmacodynamic biomarker.
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Then Sonia talks about RT-QuIC or plasma biomarker for #EarlyDiagnosis and #TrialEndPoints #solveRD... 👇 "markers of prion seeding activity & neuronal damage do not reliably cross-sectionally distinguish mutation carriers from controls" https://t.co/
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.@cureffi: What is the natural history of PrP in mutation carriers vs controls? Overall, most healthy mutation carriers seemed normal on metrics checked compared to controls. https://t.co/mCcZWqkOY8 #GRD21
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Work is being done.. The March forward never ceases.