Targeting the Receptor of Glial Cell Line-Derived Neurotrophic factor to treat Parkinson’s Disease
Objective: Our goal is to develop small molecules which support and restore nigrostriatal dopamine neurons by activation of glial cell line-derived-neurotrophic factor (GDNF) receptors. Background:…Cardiac Safety of PBT434, an Inhibitor of Alpha-Synuclein Aggregation for the Treatment of Multiple System Atrophy (MSA)
Objective: Evaluate potential cardiac liability of PBT434 using concentration-response and outlier analyses of ECG and pharmacokinetic (PK) data from a Phase 1 study of PBT434…New tool to localize seizure onset zone from sEEG signal-connectivity epileptogenicity index
Objective: The main diagnostic challenge of drug resistant epilepsies is to find epileptogenic zone, which will be enough to remove to reach seizure freedom. These…Remote monitoring of physical activity in Veterans with Parkinson’s disease
Objective: To determine the feasibility of a remote physical activity monitoring program for Veterans with Parkinson’s disease (PD). Background: Regular physical activity improves overall health,…Association Between Diet & Parkinson’s Disease Progression
Objective: The objective of this study was to evaluate whether food choices are associated with patient-reported Parkinson's disease (PD) severity over time. Background: Several traditional…Translatable biomarkers in gene therapy for Huntington’s Disease: learnings from pre-clinical studies
Objective: We have developed AAV5-miHTT, a recombinant AAV-based gene therapy expressing an engineered microRNA that specifically binds to HTT exon1, resulting in lowering of both…Exploring dopamine transporter single-photon emission computed tomography quantification as a measure of disease progression in idiopathic Parkinson’s disease
Objective: To evaluate the ability of DAT SPECT quantification to track disease progression in subjects with idiopathic Parkinson's disease (PD) by comparing baseline scans to…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…Safety, Tolerability, and Pharmacodynamic Profile of DNL201 at dose levels demonstrating LRRK2 inhibition in Parkinson’s Disease Patients with and without LRRK2 mutations
Objective: To evaluate safety, tolerability, and target engagement of DNL201, a LRRK2 inhibitor, in Parkinson’s disease (PD) patients. Background: Mutations in LRRK2 impact both familial…
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