VQ-101, a CNS-Penetrant Small Molecule Allosteric Activator of Glucocerebrosidase (GCase), Demonstrates Favorable Tolerability and Sustained Activation of Lysosomal GCase in Individuals with Parkinson’s Disease
Objective: To assess the safety, tolerability, pharmacokinetics and pharmacodynamics of VQ-101 in healthy volunteers (HVs), and individuals with Parkinson’s disease (PD) with and without GBA1…The sphingolipid Lyso-Gb3, a biomarker for the lysosomal storage disorder Fabry Disease, is significantly elevated in blood from patients with Parkinson’s Disease
Objective: To better understand lysosomal dysfunction in Parkinson’s Disease. Background: Lysosomal dysfunction and α-synuclein accumulation play central roles in Parkinson’s Disease. They are interconnected by…The Role of Autophagy Lysosomal and Ubiquitin Proteasomal System in the Pathophysiology of Parkinson’s disease.
Objective: The research how to better understand the molecular mechanism initiating the etiology of Parkinson's disease in order to appropriate the precise diagnosis and therapeutic…GCase and Ganglioside GM1 Interaction in Parkinson’s Disease
Objective: The project aims to further understand the role of the ganglioside GM1 in the relationship between GBA1 mutations and higher α-synuclein levels. Background: Among…Modeling Inflammation in GBA-Associated Parkinson’s Disease: Insights into Disease Mechanisms
Objective: This investigation centers on the role of inflammation in GBA-associated Parkinson’s disease (PD), a neurodegenerative disorder characterized by the progressive degeneration of dopaminergic neurons,…Neuroprotective Effects of ATP6V0C Overexpression in PFF Model Mice
Objective: Elucidating the role of lysosomal dysfunction due to altered stability and expression levels of V-type proton subunit ATP6V0C in the degradation and propagation of…Rescuing alpha-synuclein toxicity through neuron-specific enhancement of autophagy
Objective: In the present study, we aim to counteract regulatory and transcriptional modifiers of autophagy to enhance neuroprotective proteostasis and rescue alpha-synuclein (asyn) toxicity in…Bi-allelic BORCS5 Variants Result In A Wide Spectrum of Progressive Neurodevelopmental Disorders via Lysosomal Dysfunction
Objective: To characterize a new brain disorder associated with BORCS5 variants, elucidating their effect on lysosomal distribution and activity Background: BORCS5 encodes a subunit of…Prevalence of Parkinson’s Disease and Possible Parkinsonian Syndrome in Gaucher Disease: Data From the ICGG Gaucher Registry
Objective: To estimate the age-specific risk of Parkinson’s disease (PD) and possible parkinsonian syndrome (pPS) in Gaucher disease (GD) based on registry data. Background: While…Lysosomal network defects in parkinsonian patients carrying rare variants in lysosomal hydrolytic enzyme genes
Objective: Functional validation of rare variants of lysosomal genes potentially associated with early onset Parkinson's disease (EOPD) Background: The genetic load in EOPD is high;…
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