Loss-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.
A Phase 1B Trial in GBA1-Associated Parkinson's Disease of BIA-28-6156, a Glucocerebrosidase Activator.
CHDR
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
