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CONy 2024 | Ambroxol & other targeted therapies in Parkinson’s

K Ray Chaudhuri, MD, DSc, FRCP, FEAN, King’s College London, London, UK, shares an overview of current studies looking into targeted therapies in Parkinson’s disease. One standout example is ambroxol, a repurposed cough medicine that has been shown to act as a glucocerebrosidase (GCase) enhancer. Mutations in the GBA gene that encodes GCase are common in PD, with L444P being a notable one. Ambroxol shows promise in potentially delaying or reducing Parkinson’s symptoms such as cognitive issues, REM sleep disorders, and motor symptoms. Using ambroxol early, when the genetic mutation is detected but before Parkinson’s symptoms appear, could delay disease onset, a significant step in translating genomic theory to clinical use. Other examples of research include LRRK2 inhibitors and α-synuclein antibodies, though they’re not as advanced. This interview took place at the 18th Annual Congress on Controversies in Neurology (CONy 2024) in London, UK.

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