Safety, Tolerability, and Pharmacodynamic Profile of DNL201 at dose levels demonstrating LRRK2 inhibition in Parkinson’s Disease Patients with and without LRRK2 mutations
Objective: To evaluate safety, tolerability, and target engagement of DNL201, a LRRK2 inhibitor, in Parkinson’s disease (PD) patients. Background: Mutations in LRRK2 impact both familial…Phase I Study Design of a Leucine-Rich Repeat Kinase 2 (LRRK2) Antisense Oligonucleotide, for Parkinson’s Disease
Objective: We will present the preclinical efficacy and safety data and the design of the first in human trial of an antisense oligonucleotide (ASO) for…Longitudinal clinical and imaging characteristics of non-manifest LRRK2 carriers: The PPMI cohort
Objective: To examine longitudinal change in the clinical characteristics of non-manifesting LRRK2 mutation carriers (NMCs) compared to healthy controls (HC) in the Parkinson’s Progression Markers…Poly-ADP-ribose polymerase 1 modulates mitochondrial fission via LRRK2 in a rotenone-induced Parkinson’s disease model
Objective: To clarity the effects and mechanism of poly-ADP-ribose polymerase 1(PARP1) in regulating mitochondrial fusion/fission in a rotenone-induced Parkinson's disease (PD) model. Background: Accumulating evidences…First clinical and genetic analysis of LRRK2 G2019S, R1441G/C/H/, I2020T and Y1699C mutations in Costa Rican patients with Parkinson’s disease
Objective: To assess the frequency of G2019S, R1441G/C/H, I2020T and Y1699C mutations in a Costa Rican cohort of Parkinson’s disease (PD) cases and controls. Background:…Virtual Assessment of LRRK2 Carriers to Optimize Research in Parkinson Disease (VALOR-PD): A Comparison to Traditional Cohorts
Objective: To compare a national virtual cohort of LRRK2 G2019S carriers, identified via a personal genetics company, to existing traditional cohorts. Background: Advancements in personal…Rare variants of the LRRK2 gene in the haplotype as one of the potential risk factors for endemic parkinsonism in a small isolated region in the Czech Republic
Objective: The aim of the study was to investigate a co-occurence of rare variants in set of genes frequently associated with parkinsonism. Background: The increased…Interest of multiplex ligation-dependent probe amplification assay to identify pathogenic mutations in Parkinson’s disease patients
Objective: To evaluate the interest of the MLPA (Multiplex Ligation-dependent Probe Amplification) commercially available kit in the genetic testing in Parkinson’s disease (PD). Background: PD…LRRK2 p.G2019S and p.N2081D variants as modifiers of glucocerebrosidase activity
Objective: To examine whether the activity of the lysosomal enzyme glucocerebrosidase (GCase) is associated with LRRK2 variants. Background: Mutations in GBA (which encodes GCase) and…Genetic study of patients with Parkinson’s disease subjected to second line therapies
Objective: Parkinson's disease (PD) is a neurodegenerative disorder characterized by remarkable phenotypic variability. Accumulated evidence points that the manifestation of PD clinical signs might be…
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