Transitional Life Events in Friedreich Ataxia: Differential Age at Onset Perspectives
Objective: To identify and quantify the age-contingent impact of Friedreich’s Ataxia. Background: Friedreich's ataxia (FA) is a rare, inherited multisystem neurodegenerative disorder that primarily affects…Modeling disease progression in spinocerebellar ataxias
Objective: In the context of emerging gene-targeted therapies for polyglutamine spinocerebellar ataxias (SCAs), identifying predictive clinical markers of disease progression is becoming increasingly relevant. We…Prasinezumab in Early-Stage Parkinson’s Disease: Additional Data from the PADOVA Study
Objective: To evaluate the effect of prasinezumab on delaying disease progression in participants with early-stage Parkinson’s disease (PD) on stable symptomatic medication. Background: Prasinezumab is a humanised IgG1…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…Clinical Drug-Drug Interaction Profile Of BIA 28-6156: Results From Three Independent Phase I Studies
Objective: Three independent, open-label, clinical drug-drug interaction (DDI) studies were conducted to characterise the clinical DDI profile of BIA 28-6156. Background: BIA 28-6156 (paricerat) is…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)…
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