medsynapse-hcp
Introduction Gaucher disease type 3 (GD3) is a genetic, progressive lysosomal storage disorder characterized by visceral manifestations and chronic neurologic symptoms including horizontal ophthalmoplegia/supranuclear gaze palsy, ataxia and dystonia (1). Existing therapies like enzyme replacement (ERT) and substrate reduction therapy (SRT) are effe
Brain-Penetrant Therapy in GD3: A Case Study on Venglustat and Imiglucerase Combination

Brain-Penetrant Therapy in GD3: A Case Study on Venglustat and Imiglucerase Combination

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