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A Phase 1B Trial in GBA1-Associated Parkinson's Disease of BIA-28-6156, a Glucocerebrosidase Activator.
1 July 2023. doid: 10.1002/mds.29346
den Heijer JM, Kruithof AC, Moerland M, Walker M, Dudgeon L, Justman C, Solomini I, Splitalny L, Leymarie N, Khatri K, Cullen VC, Hilt DC, Groeneveld GJ, Lansbury P
View publicationLoss-of-function mutations in the GBA1 gene are one of the most common genetic risk factors for onset of Parkinson's disease and subsequent progression (GBA-PD). GBA1 encodes the lysosomal enzyme glucocerebrosidase (GCase), a promising target for a possible first disease-modifying therapy. LTI-291 is an allosteric activator of GCase, which increases the activity of normal and mutant forms of GCase.
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