Overview
- The 12-month, placebo-controlled trial in 55 patients confirmed that daily Ambroxol was safe, well-tolerated and achieved therapeutic concentrations in the brain.
- Participants on Ambroxol maintained stable neuropsychiatric profiles while those on placebo experienced worsening psychiatric symptoms.
- Patients carrying high-risk GBA1 gene variants showed measurable cognitive improvements when treated with Ambroxol.
- GFAP levels, a blood marker of glial cell damage, rose in the placebo group but remained steady in Ambroxol recipients, indicating potential neuroprotective action.
- A larger, dedicated trial focusing specifically on cognitive outcomes is scheduled for later in 2025 to further evaluate Ambroxol’s disease-modifying potential.