The Corticobasal Degeneration Functional Rating Scale (CBD-FS)
Objective: To develop a patient/care giver-reported scale that is capable of assessing functional disability in CBD that can be easily and reliably used in clinical…WTX101 – A Novel Copper Modulating Agent for Wilson Disease Demonstrates Efficacy and Safety in a Phase 2, Multi-Center, Open Label Study
Objective: The objective of this study was to evaluate the efficacy and safety of 24 week WTX101 treatment in newly diagnosed Wilson Disease (WD) patients. Background:…Human-derived α-synuclein antibody BIIB054 binds pathologic forms of α-synuclein and attenuates transmission of α-synuclein in vitro and in vivo
Objective: To identify and characterize a human antibody against α-synuclein (α-syn) for the clinical development of an immunotherapy for Parkinson's disease (PD). Background: α-syn plays…Novel small molecules targeting alpha-synuclein aggregation for the treatment of Parkinson’s disease
Objective: To develop novel, small molecular weight compounds, that can interfere with the aggregation process of alpha-synuclein (aSyn), as disease modifying agents. Background: The aggregation…Critical path for Parkinson’s, I: Data sharing and regulatory science in catalyzing innovation for Parkinson’s disease
Objective: This presentation highlights a new precompetitive consortium based strategy for the successful development of regulatory-endorsed biomarkers and quantitative drug development platforms targeted at the…The Parkinson progression marker initiative (PPMI) – Developing a comprehensive longitudinal biomarker dataset
Objective: The goal of the Parkinson Progression Marker Initiative (PPMI) is to identify clinical, imaging and biospecimen biomarkers of PD progression to provide tools for…Multi-modal recruitment strategy leads to expeditious enrollment in STEADY-PD III
Objective: To review a successful multi-modal recruitment strategy in a clinical trial of early untreated PD patients. Background: STEADY-PD III is a multicenter NINDS funded…Deep brain stimulation in early stage Parkinson’s disease: Is it disease modifying?
Objective: To examine patterns of motor symptom worsening in the 2 years following deep brain stimulation (DBS) in early stage Parkinson's disease (PD). Background: Although…Evaluation of regional changes in striatal DAT binding in a progressing Parkinson’s disease (PD) cohort: Lessons for imaging biomarker assessment in disease-modifying therapeutic trials
Objective: To evaluate subregion-dependent striatal changes in dopamine transporter (DAT) binding using 123-I Ioflupane SPECT in PD participants in the Parkinson Progression Marker Initiative (PPMI)…Fifteen years of clinical trials in Huntington’s disease: Too many clinical trial failures
Objective: To investigate the trends in Huntington's disease (HD) therapeutic development by analyzing interventional clinical trials publicly registered for the last 15 years. Background: Like…
Abstracts from the International Congress of Parkinson’s and Movement Disorders.