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MDS 2023 | The potential of ambroxol in Parkinson’s disease dementia: Phase II trial results

Stephen Pasternak, MD, PhD, FRCPC, Robarts Research Institute, Western University, London, Ontario, Canada, discusses a trial exploring the potential of ambroxol, an over-the-counter cough medicine, in the treatment of Parkinson’s disease dementia (PDD). Despite not meeting the primary endpoints, the trial revealed some promising signals. The trial enrolled a diverse group of patients, which contributed to the challenges faced in achieving conclusive results. However, patients with GBA mutations appeared to respond better to ambroxol. Further analysis is required to understand the impact of blood and spinal fluid biomarkers on treatment response. With a lack of disease-modifying options for PDD, the safe and widely available nature of ambroxol suggests the need for continued development. This interview took place at the 2023 International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen, Denmark.

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