Product Description
LTI-291 is a small-molecule activator of glucocerebrosidase (GCase), a lysosomal enzyme involved in the breakdown of glycosphingolipids. Mutations in GBA1, the gene encoding GCase, are the leading genetic risk factor for the synucleinopathies Parkinson's disease and dementia with Lewy bodies. GBA1 mutations that lower enzyme activity are associated with a higher risk of developing PD, more severe disease, and faster progressing disease. (Sourced from: https://www.alzforum.org/therapeutics/lti-291)
Mechanisms of Action: Gcase Activator
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: N/A
FDA Designation: *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: None
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Company Founding Year: None
Additional Commercial Interests: None
Clinical Description
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