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Clinical trials of drugs targeting GBA-PD
| Drug | Pharmacological group | Mechanism | Phase |
|---|---|---|---|
| Ambroxol | Pharmacological chaperone | Activation of GCase | II |
| Venglustat (GZ/SAR402671) | Substrate reduction therapy | A decrease in the substrate concentration (inhibition of glucosylceramide synthase) | II |
| LTI-291 | Pharmacological chaperone | Allosteric activator of GCase | Ib |
