The Allosteric Activator of Lysosomal Glucocerebrosidase VQ-101 Demonstrates Robust Evidence of Target and Pathway Engagement in People with Parkinson’s Disease: Results from a 6-Month Open-Label Extension Study
Objective: To evaluate the continued safety, pharmacokinetics (PK), and pharmacodynamics of VQ-101 in patients with Parkinson’s disease (PD) with and without a GBA1 mutation. Background:…Parkinsonism Revealing a Late Form of Niemann-Pick Disease About a Case
Objective: Niemann-Pick type B (NPB) is a rare, autosomal dominant hereditary disorder. Clinical presentation is primarily visceral. Neurological manifestations are rare, unlike in other types…Genetically proven Juvenile-Onset Niemann–Pick Disease Type C Presenting with Dystonia- Ataxia syndrome.
Objective: We present a case Juvenile-Onset Niemann–Pick Disease Type C (NPC) who Presented to us with progressive Dystonia- Ataxia syndrome. Background: Niemann–Pick disease type C…Characterizing Movement Disorders in Niemann-Pick Disease Type C -A Registry-Based Analysis with Findings from the International Niemann-Pick-Disease Registry (INPDR)
Objective: We aim to define the prevalence and phenotypic spectrum of movement disorders in Niemann-Pick Disease Type C (NPC), track motor and functional progression using…CSM-101 is a Small Molecule Agonist of TRPML1 for Parkinson’s-Related Disorders
Objective: (i) To develop a brain-penetrant small molecule agonist for the lysosomal TRPML1 channel and, (ii) Perform preclinical studies supporting development in Parkinson’s-related disorders. Background:…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,…
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