Overview

• In a 12-month randomized trial of 55 patients, Ambroxol was safe, well-tolerated and achieved therapeutic brain levels.
• Patients on Ambroxol maintained stable GFAP levels while the placebo group experienced increases linked to brain cell damage.
• Neuropsychiatric symptoms worsened under placebo but remained stable among those receiving Ambroxol.
• Participants with high-risk GBA1 gene variants demonstrated cognitive improvements on Ambroxol compared to placebo.
• Researchers plan a follow-up cognitive endpoints trial later this year and await regulatory approval in Canada and the United States.