Clinical and Genetic Profile of the ACTIVATE Population: A Phase 2 Trial of BIA 28-6156 in GBA-PD
Objective: To characterize the patient population enrolled in the ACTIVATE study, a Phase 2 multicenter, randomized, double-blind, placebo-controlled trial assessing the efficacy, safety, tolerability, pharmacodynamics,…Prospective Comparison of Volumetric and Diffusion MRI Biomarkers for Multiple System Atrophy
Objective: We aimed to develop new imaging biomarkers allowing objective monitoring of early Multiple System Atrophy (MSA) progression, which could improve outcome definition for future…Physical Activity is Associated with Attenuated Motor and Cognitive Progression in Parkinson’s Disease
Objective: To determine whether physical activity (PA) modifies the rate of motor and cognitive progression in Parkinson’s disease (PD) and to identify specific components of…Continuous Subcutaneous Foslevodopa/Foscarbidopa After Deep Brain Stimulation or Levodopa–Carbidopa Intestinal Gel: A Case Series in Advanced Parkinson’s Disease
Objective: To describe indications and short-term outcomes of FLC/FCD in PD patients previously treated with DBS and/or LCIG. Background: Advanced Parkinson's disease patients treated with…A Phase 1b Study to Investigate Safety, Tolerability and Biomarker Responses of Repeated Subcutaneous Doses of HER-096 in Subjects with Parkinson’s Disease
Objective: The aim of this study was to investigate safety, tolerability and biomarker responses of repeated subcutaneous (s.c.) doses of HER-096 in subjects with Parkinson’s…Inpatient Titration Protocol and Practical Implementation of Continuous Subcutaneous Foslevodopa/Foscarbidopa: Lessons from 28 Consecutive Cases
Objective: To describe inpatient titration methodology, dosing parameters, support services utilization, and early challenges in consecutive advanced PD patients. Background: Successful implementation of continuous subcutaneous…SNCA-Targeting RNAi Therapeutics Prevent Disease Progression in a Synucleinopathy Mouse Model and Enable Robust, Prolonged, and Safe Alpha-Synuclein Protein Reduction in the Cynomolgus Monkey Central Nervous System
Objective: Assess the nonclinical efficacy and safety of C16-conjugated siRNAs targeting SNCA mRNA (SNCA siRNA). Background: Parkinson’s disease is a neurodegenerative disorder with an estimated…Disease-Modifying Outcomes Following High Intensity Boxing and Sensory Exercise in Parkinson’s Disease: Results From a Double-blinded Randomized Controlled Trial
Objective: The purpose of this study was to determine whether high-intensity exercise (boxing) and sensory exercise (PD SAFEx™) produce disease-modifying effects on motor symptoms following…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…
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