Lysosomal Storage Disorders Research

Review finds limited evidence for ambroxol in Gaucher and genetic Parkinson disease

A systematic review examined clinical research on ambroxol in people with Gaucher disease and GBA1-related Parkinson disease. It found some reported biochemical and neurological changes in selected patients, but the studies were generally small, uncontrolled and varied substantially. The review concluded that ambroxol remains investigational and that its long-term safety and clinical effectiveness are uncertain.