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Ambroxol Trial Confirms Safety, Brain Delivery and Early Cognitive Benefits in Parkinson’s Dementia

Phase 2 results showing stable biomarkers with improved cognition in genetically at-risk patients pave the way for a larger cognition-focused study later this year.

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This cough medicine is an unlikely new ally in slowing Parkinson's disease dementia
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Overview

  • A 12-month placebo-controlled trial of once-daily Ambroxol in 55 patients with Parkinson’s disease dementia confirmed its safety and ability to reach targeted brain concentrations.
  • Ambroxol recipients maintained stable neuropsychiatric symptoms while those on placebo experienced worsening psychiatric scores.
  • Levels of GFAP, a blood marker of brain cell damage, remained stable in the Ambroxol group compared with increases among placebo patients.
  • Participants with GBA1 gene variants exhibited preliminary improvements on cognitive assessments when treated with Ambroxol.
  • Although Ambroxol is approved in Europe for respiratory use and lacks clearance in North America, funding from the Garfield Weston Foundation supports a larger cognition-focused trial to begin later this year.