Article
A Phase 1B Trial in GBA1-Associated Parkinson's Disease of BIA-28-6156, a Glucocerebrosidase Activator.
Movement disorders : official journal of the Movement Disorder Society - 1 Jul 2023
den Heijer Jonas M, Kruithof Annelieke C, Moerland Matthijs, Walker Mike, Dudgeon Lindsay, Justman Craig, Solomini Imelda, Splitalny Leslie, Leymarie Nancy, Khatri Kshitij, Cullen Valerie C, Hilt Dana C, Groeneveld Geert Jan, Lansbury Peter
Abstract excerpt
BACKGROUND: 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....
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