REGENERATE-PD: A Phase 2, Randomized, Double-Blind, Surgery-Controlled Study of GDNF Gene Therapy (AAV2-GDNF; AB-1005) for Moderate Stage Parkinson’s Disease
Objective: To present the REGENERATE-PD study design: a phase 2, randomized, double-blind, surgery-controlled trial assessing efficacy and safety of intraputaminal adeno-associated virus serotype 2 containing…Plasma microRNA expression profiling in patients with progressive supranuclear palsy. A five-microRNA panel in plasma was identified as a potential biomarker for progressive supranuclear palsy
Objective: Our objective was to identify differentially expressed, circulating miRNAs in PSP which could serve as diagnostic biomarker for this disease. Background: Progressive supranuclear palsy…Phase I-II first-in-human clinical trial of intraputamenal CDNF in Parkinson’s disease: Topline results of the six-month double-blind placebo-controlled main study and the six-month active treatment extension study
Objective: To assess the safety and tolerability and to explore the preliminary efficacy of intermittent intraputamenal monthly infusions of CDNF in subjects with advanced idiopathic…Phase I-II first-in-human clinical trial of intraputamenal CDNF in Parkinson’s disease: Exploratory fluid-based biomarker endpoints of the 12-month treatment period
Objective: To explore fluid-based biomarkers in subjects with advanced idiopathic Parkinson’s disease (PD) before and after intermittent intraputamenal monthly infusions of CDNF. Background: CDNF is…Phase I-II first-in-human clinical study of intraputamenal CDNF in Parkinson’s disease: Exploratory PET imaging endpoints of the 12-month treatment period
Objective: To test if intermittent intraputamenal monthly infusions of CDNF have an effect on striatal dopamine transporter (DAT) binding, as measured by PET, in subjects…Effect of CDNF on non-motor symptoms like motivation, fear, stress and depression-like behavior
Objective: The goal of this study was to characterize the effect of CDNF on motivation, stress and depression-like behavior, because most of the non-motor symptoms,…AAV-mediated over-expression of VEGF-B in PINK1 gene knockout rats increases striatal dopamine content
Objective: To evaluate overexpression of the neuroprotective vascular endothelial growth factor B (VEGF-B) in a genetic Parkinson’s disease (PD) model. Background: We have shown in…DBS Plus: Investigating cell therapy and deep brain stimulation as an approach to alter disease progression and treat motor and non-motor symptoms of Parkinson’s disease
Objective: Investigate safety, feasibility, and potential efficacy of cell therapy combined with DBS to treat and modify symptoms and progression of PD. Background: Our strategy…Serum brain-derived neurotrophic factor as an indication of disease state in parkinsonism and RBD
Objective: Evaluation of peripheral concentrations of brain-derived neurotrophic Factor (BDNF) in patients with Parkinsonian disorders and correlation with clinical measurements of disease progression to elucidate…Pathological findings from two Parkinson’s disease patients after intraputaminal GDNF infusion
Objective: To describe the postmortem findings from two patients who underwent intraputaminal infusion of glial-derived neurotrophic factor (GDNF). Background: GDNF has been shown to have…
Abstracts from the International Congress of Parkinson’s and Movement Disorders.