Diagnostic mismatches between clinical assessment, imaging and alpha-synuclein biomarkers in parkinsonian syndromes
Objective: To analyze the frequency of matching information provided by traditional clinical assessment, dopamine transporter (DaTscan), and skin biopsy results in patients with undiagnosed parkinsonian syndromes. Background:…Parkinson’s Progression Markers Initiative (PPMI): A Biomarker Engine for Therapeutic Development for PD and Related Synucleinopathies
Objective: PPMI is a longitudinal, observational study investigating clinical, imaging, genetic and biofluid biomarkers to accelerate therapeutic development for Parkinson’s disease (PD) and related synucleinopathies…The neuroprotective effects of Echinacoside on α-synuclein fibril-induced Parkinson’s disease mice model
Objective: This study further investigates the ameliorative effects of ECH on neurobehavioral abnormalities and nigrostriatal dopaminergic system impairment in α-synuclein preformed fibrils (α-Syn PFFs)-induced PD…Three Biopsies Optimizes Test Performance for the Detection of Cutaneous Phosphorylated Alpha-Synuclein
Objective: To review two independent data sets to define the test performance of skin biopsies for the detection of P-SYN and peripheral nerve degeneration in…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…First in Class ASO Targeting A53T Allele: Preclinical Efficacy
Objective: Our objective was to develop an antisense oligonucleotide (ASO) allele-specific strategy to target the mutant A53T SNCA allele, reducing aberrant α-synuclein production, without downregulating…Relationship Between Alpha-Synuclein Aggregation Profiles, Imaging Biomarkers, and Disease Severity in a Phase 2 Study of ATH434 in MSA
Objective: To investigate the relationships between α-synuclein aggregation profiles, imaging biomarkers, and UMSARS I scores in the ATH434-201 Phase 2 study. Background: Multiple system atrophy…A Novel Therapeutic Targeting the tPA-NMDAr Interaction for Parkinson’s Disease
Objective: Glunomab, a monoclonal antibody designed to inhibit the interaction between tissue plasminogen activator (tPA) and the N-methyl-D-aspartate receptor (NMDAr), has demonstrated therapeutic potential across…Organoid-Based AI Clinical Trial Platform for Phase 0 Studies in Parkinson’s Disease
Objective: we developed an organoid-based AI-driven platform to classify patient specific molecular subtypes and predict drug efficacy through an AI-powered validation pipeline. Patient-derived induced pluripotent…Plasma Biomarkers Predict Clinical Outcomes in Individuals with Neuronal α-Synuclein Disease and Amyloid-β Co-Pathology
Objective: We examined whether the plasma biomarkers pTau181, pTau217, GFAP, NfL, and Aβ42/40 are associated with cognitive, functional, and motor outcomes in individuals with neuronal…
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