Human Snca Knock-In Mouse as a Model for Synucleinopathy Research
Objective: Inter-species differences between mouse and human α-synuclein (asyn) may influence aggregation and pathology in animal models, highlighting the need for models expressing human asyn.…Long-Term Open-Label Data on the GCase Modulator GT-02287 in People with Parkinson’s Disease
Objective: To evaluate the long-term safety, tolerability, and PK of the oral, brain-penetrant glucocerebrosidase (GCase) modulator GT-02287 in people with Parkinson’s disease. Background: Preclinical evidence…Pattern and progression of sudomotor dysfunction in alpha synucleinopathies
Objective: This study examined the distribution and longitudinal progression of sweat loss in patients with PAF. Background: Pure autonomic failure (PAF) is an alpha-synucleinopathy with…α-Synuclein biomarkers in prodromal synucleinopathies: a systematic review and meta-analysis.
Objective: To quantify the diagnostic accuracy and predictive performance of α-synuclein biomarkers in isolated REM sleep behaviour disorder (iRBD), pure autonomic failure (PAF), and idiopathic…Clinical and Biomarker Trajectories in GBA1 Non-Manifesting Carriers With and Without α-Synuclein Seed Amplification Positivity
Objective: To characterize clinical and biomarker features of GBA1-NMC and evaluate whether CSF-αSynSAA status is associated with longitudinal clinical progression and phenoconversion. Background: Pathogenic variants…Kinetic Features of Alpha-synuclein Positivity in CSF Seed Amplification in Parkinson’s Disease: an Exploratory Cross-cohort Analysis
Objective: To evaluate whether kinetic signatures from alpha-synuclein CSF seed amplification assays (aSynSAA) are generalizable among SAA-positive Parkinson’s Disease (PD) samples across previously published cohorts.…Uncovering Parkinson Disease Progression Biomarkers with Plasma Proteomics and Functional Genomics
Objective: This study's objective was to discover novel plasma protein biomarkers related to progression and treatment response with a longitudinal PD cohort using an integrated…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:…Beyond α-Synuclein SAA: A Multimodal Minimally Invasive Platform for Biologically Informed Diagnosis of Parkinsonian Syndromes
Objective: To evaluate whether integrating dermal α-synuclein and 4R-tau seed amplification assays (SAAs) with serum neurofilament light chain (NfL) improves diagnostic precision in neurodegenerative parkinsonian…Pain Management in Parkinson’s Disease: The Role of TRPV1 as a Therapeutic Target
Objective: Parkinson’s Disease (PD) is the second most common neurodegenerative disorder. Pain is one of the most prevalent non-motor symptoms in PD, impacting health-related quality…
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