Phase 1 Study of BT-267, a potent, selective, brain-penetrant, and oral small molecule inhibitor of LRRK2
Objective: The aim of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of BT-267 in healthy adults. Background: Leucine rich repeat kinase…SAA Positivity Rate Amongst Dual LRRK2-GBA1, GBA1 and LRRK2 Carriers with Parkinson’s Disease
Objective: We investigated positivity rate in dual GBA1+LRRK2-PD by comparing GBA1+LRRK2-PD carriers(n=13), GBA1-PD(n=169) and LRRK2-PD(n=175). Background: GBA1 and LRRK2 variants represent the most common genetic…Preliminary Genetic Spectrum of Parkinson’s Disease in the Israeli PD GENEration Cohort
Objective: To characterize the preliminary genetic spectrum of the Israeli PD GENEration cohort. Background: The PD GENEration study (NCT04994015) provides genetic testing and counseling for…Sex-Specific Associations of APOE ε4 with Cognitive Decline in Parkinson’s Disease: Insights from the Critical Path Institute GEM-PD Initiative
Objective: We aim to advance precision medicine strategies for Parkinson’s disease (PD) through data-driven research led by the Critical Path for Parkinson’s (CPP) global public-private…Motor Fluctuations and Dyskinesias in LRRK2-Associated Parkinson’s Disease
Objective: To characterize motor fluctuations and dyskinesias in patients with LRRK2-associated Parkinson’s disease. Background: Motor complications, including dyskinesias and motor fluctuations, are hallmarks of advanced…LRRK2 Mutation Spectrum and Association Study in a Multi-ethnic Cohort of Malaysian Parkinson’s Disease Patients
Objective: To systematically characterize the spectrum, haplotypes, and cumulative burden of LRRK2 variants and determine their associations with PD risk in the multi-ancestry Malaysian population.…Predictors of Parkinson’s Disease Phenoconversion in Non-manifesting LRRK2 Carriers
Objective: To determine clinical predictors of Parkinson’s disease (PD) phenoconversion in a prodromal LRRK2 carrier cohort. Background: Non-manifesting carriers of LRRK2 mutations represent an important…Bisphenol S: A Potential Environmental Modifier of LRRK2-Associated Parkinson’s disease
Objective: To validate candidate environmental modifiers in the LIPAD exposomics study using additional cell lines, and test a proposed “gene-mitochondria-environment axis” in regulating LRRK2-Parkinson's disease…LRRK2 G2019S Drives Lysosomal Lipid Remodeling and Inflammation in Human iPSC-Derived Microglia
Objective: The aim of this study is to elucidate disease-related metabolic and inflammatory pathways driven by LRRK2 G2019S in human iPSC-derived microglia. Background: Parkinson’s Disease…Brain-targeted dual-loaded extracellular vesicles enable PROTAC-mediated LRRK2 degradation in Parkinson’s disease
Objective: To develop a brain-targeted extracellular vesicle (EV) platform capable of delivering proteolysis-targeting chimeras (PROTACs) across the BBB and inducing degradation of pathogenic proteins in…
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