Fatty acids and Glucocerebrosidase activity as potential biomarkers of stearyl-CoA desaturase inhibition; an observational study of inter and intraday variation in healthy volunteers and patients with Parkinson’s disease
Objective: This study aimed to assess the naturally occurring levels and variation in plasma fatty acids (FA) and ceramide metabolism in healthy volunteers (HV) and…Effects of psychiatric tests on disease severity and nonmotor symptoms in early onset Parkinson’s Disease
Objective: Psychiatric tests evaluating symptoms such as depression, anxiety, rumination, obsession, impulsivity, apathy and impulsive compulsive behaviors were applied in patients with early onset PD.…Wearable sensors are able to identify individuals in the prodromal phase of X-linked Dystonia-Parkinsonism
Objective: To identify subclinical changes of balance and gait in patients with X-linked dystonia-parkinsonism (XDP) and non-manifesting carriers of the XDP-causing mutation. Background: XDP is…Falls in people with typical Parkinson’s disease and the secret of unexpected functional mobility. A protocol for a mixed-method study design.
Objective: Understand why some people with typical Parkinson's disease (PwP) show unexpected functional mobility e.g. functional mobility despite the presence of risk factors. Consequently, the…Homeostatic Improvement of Brain Bioenergetic Metabolism in Parkinson’s Disease: Results From A Phase 2 REPAIR-PD Clinical Trial With CNM-Au8
Objective: To determine the effects of the nanocatalytic drug, CNM-Au8, on brain energy metabolites in Parkinson’s Disease. Background: Converging lines of evidence are identifying brain…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…
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