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Estrone is metabolized further into intermediate compounds 2-hydroxyestradiol (2-OH-E1) and 4-hydroxyestradiol (4-OH-E1)

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A number of approaches targeting deficient GCase activity in GBA-PD have reached the stage of clinical phase 2/3 trials including orally active GCase chaperones to enhance enzymatic activity, substrate reduction therapies modelling current treatment of Gauchers disease and GBA gene therapy (Table 5)