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…Alpha-Synuclein Accumulation-Attenuating Effect of Brain-Derived Neurotrophic Factor in Primary Cell Culture Model of Parkinson’s Disease
Objective: To explore the role of neurotrophic signaling pathways in modulating the effect of brain-derived neurotrophic factor (BDNF) on pre-formed fibrils (PFFs)- induced alpha-synuclein (aSyn)…Parkinson’s disease (PD) associated with the E46K mutation in the SNCA gene, twenty years after
Objective: To provide an updated overview of Parkinson’s disease (PD) associated with E46K mutation carriers in the SNCA gene 20 years after its description, incorporating…Cutaneous Phosphorylated Alpha-Synuclein Deposition in Multiple System Atrophy
Objective: To report differences in the amount, localization and topographic distribution of cutaneous P-SYN deposition between cerebellar and parkinsonian variants of MSA. Background: MSA is…Results From a Dose-Blinded, Phase II Extension Study Evaluating Long-Term Efficacy, Safety and Tolerability of Minzasolmin in Participants With Parkinson’s Disease
Objective: ORCHESTRA XT (PD0055; NCT05543252) was a Phase II, dose-blinded extension of the ORCHESTRA Phase II, proof-of-concept study (NCT04658186). ORCHESTRA XT assessed the pharmacodynamic effects of…Serum based estimations of CFAH, Gelsolin, CNDP1 and DKK3 levels suggest involvement of multiple pathways in pathogenesis of Parkinson’s disease.
Objective: To estimate levels of complement factor H (CFAH), phosphorylated alpha-syn (p- a-syn), gelsolin, carnosine dipeptidase-1 (CNDP1) and DKK3 in serum of Parkinson’s disease (PD) patients…
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