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Defective Activity of GCase Enzyme Linked with Neurodegeneration in Parkinson’s, Mouse Study Finds

Impaired activity of the enzyme glucocerebrosidase (GCase), which is responsible for breaking down and recycling cell waste, boosts neurodegeneration and accumulation of alpha-synuclein in Parkinson’s, according to a new mouse study. The research, “Development and biochemical characterization of a mouse model of Parkinson’s disease bearing defective…

First Patient Dosed with VY-AADC Gene Therapy in Parkinson’s Phase 2 Trial

Voyager Therapeutics has begun patient dosing in a Phase 2 trial testing its investigational VY-AADC gene therapy in Parkinson’s patients whose motor symptoms are not responding adequately to oral medication. The trial, called RESTORE-1 (NCT03562494), is recruiting participants across seven sites in the United States. VY-AADC is a therapy that delivers…