Loss-of-function mutations in the GCase gene, GBA1, are the leading genetic risk factor for the synucleinopathies Parkinson’s disease and dementia with Lewy bodies (DLB). Interest in repurposing ambroxol to treat Parkinson’s disease stems from its activity as a molecular chaperone for the lysosomal enzyme glucocerebrosidase (GCase) ( Maegawa et al., 2009). Ambroxol is unavailable in North America. The most common side effects are gastrointestinal. Syrup, tablet, and other forms are used at doses of 30 to 120 mg per day to reduce or thin mucous secretions and ease sore throat pain. BackgroundĪmbroxol is a mucolytic compound and the main ingredient of over-the-counter cough medicines sold in many countries. FDA Status: Parkinson's Disease (Phase 2), Parkinson's Disease Dementia (Phase 2), Dementia with Lewy Bodies (Phase 2) Status in Select Countries: OTC for cough relief. Name: Ambroxol Synonyms: Ambrosan, Mucosolvan Chemical Name: trans-4-(2-Amino-3,5-dibrombenzylamino)-cyclohexanol Therapy Type: Small Molecule (timeline) Target Type: alpha-synuclein, Other (timeline) Condition(s): Parkinson's Disease, Parkinson's Disease Dementia, Dementia with Lewy Bodies U.S. How would you like to share? Facebook Twitter LinkedIn
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