| Molecular chaperones | Small molecules that can stabilize mutant glucocerebrosidase and enhance residual enzyme activity |
| Gene therapy | Viral vector delivery of functional GBA gene to increase glucocerebrosidase expression |
| Substrate reduction | Pharmacological approaches to reduce glucosylceramide accumulation |
| Autophagy enhancers | Compounds that can improve lysosomal function and clearance of alpha-synuclein |
| Databases | OMIMOrphanetClinicalTrialsPubMed |
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