Results from a Randomized, Double-Blind, Placebo-Controlled Study of ATH434 in MSA using CSF NfL as a Covariate
Objective: Evaluate efficacy and biomarker response of ATH434 in MSA, including CSF neurofilament light chain (NfL), a prespecified but previously unreported analysis using this disease-severity…A Novel Therapeutic Targeting the tPA-NMDAr Interaction for Multiple System Atrophy
Objective: LYS241, a first-in-class, humanized, and optimized monoclonal antibody, designed to inhibit the interaction between tissue plasminogen activator (tPA) and N-methyl-D-aspartate receptor (NMDAr), has demonstrated…Prasinezumab’s Impact on Neuromelanin- and Iron-Sensitive MRI Biomarkers in Parkinson’s disease: Findings From the PADOVA Phase IIb Study
Objective: To evaluate the effects of prasinezumab on neuroimaging biomarkers in individuals with early-stage Parkinson’s disease (PD) on stable symptomatic medication. Background: PD is characterized by the…First-in-Human Single-Ascending-Dose Trial of ALN-SNCA, an Intrathecally Administered siRNA for Reducing α-synuclein, in Patients with Early-Stage Parkinson’s Disease
Objective: Development of a Phase 1, First-in-Human (FIH) clinical trial evaluating ALN-SNCA, a novel, intrathecally (IT) delivered small interfering RNA (siRNA) targeting SNCA mRNA to…Bridging the Genomic Gap: Ancestry-Aware Polygenic Risk and Variant Discovery in African Parkinson’s Disease Cohorts
Objective: This study evaluates the shift from Euro-centric data to a diversified landscape. We aim to quantify the performance of European-derived Polygenic Risk Scores (PRS)…Translational Target Engagement of HER-096 Demonstrates Concordant Proteostasis and Mitochondrial Modulation in Preclinical Models and Patients with Parkinson’s Disease
Objective: To evaluate translational target engagement of HER-096, a CDNF-derived peptidomimetic, in preclinical models of Parkinson’s disease (PD) and in a Phase 1b clinical study…12-week Aerobic Interval Training Induces Hemisphere-Specific Neuroprotection in Parkinson’s Disease: A Multimodal MRI, [18F]Fluorodopa PET/CT and BDNF Study
Objective: To investigate whether a 12-week aerobic interval training (AIT) can preserve subcortical brain structures in early Parkinson’s disease (PD) and whether these effects are…Modeling Parkinson’s Disease Progression to Quantify Long-Term Treatment Effects via the Concept of ‘Time Saved’
Objective: Evaluate the long-term effectiveness of potential disease-modifying treatments (DMTs) in Parkinson’s disease (PD) using a modelling-based virtual comparator and explore patient-relevant benefits through the…The Emerging Landscape of Genotype-Stratified Clinical Trials in Parkinson’s Disease and Atypical Parkinsonian Disorders
Objective: To characterize the landscape of genotype-stratified clinical trials in Parkinson’s disease (PD) and atypical parkinsonian disorders (APD), including therapeutic targets, geographic representation, and trial…NLRP3 Inhibition in Early-Stage Parkinson’s disease: Results of a Phase Ib Study
Objective: To evaluate the effect of selnoflast, an NLRP3 inhibitor, in persons with early-stage Parkinson’s disease (PD). Background: Damage-associated molecular patterns and pathological alpha-synuclein from damaged or…
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