Designing a multi-arm multi-stage trial for Parkinson’s
Objective: The Edmond J Safra ACT-PD initiative (EJS ACT-PD) is developing the first multi-arm multi-stage (MAMS) platform trial for potential disease modifying approaches for Parkinson’s…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…Validation of biomarkers in Huntington’s disease to support the development of disease-modifying therapies: a systematic review and critical appraisal scheme
Objective: To review studies for disease progression biomarkers published in HD and evaluate their methodological quality in a standardized manner. Background: Huntington disease (HD) is a fatal autosomal dominant neurodegenerative condition that starts to manifest clinically around the 4th decade of life. There are promising novel genetic-based therapies under development intended to modify the disease trajectory. Valid…Azathioprine immunosuppression and disease modification in Parkinson’s disease (AZA-PD): a randomised double-blind placebo-controlled phase II clinical trial
Objective: To design a clinical trial to test the concept that the immune system is integral in the progression of Parkinson’s disease (PD) and establish…FIRST-IN-MAN CLINICAL TRIAL OF INTRAPUTAMENAL CDNF IN PARKINSON’S DISEASE FINDS A CONSORTED BIOMARKER RESPONSE IN A SUBGROUP OF SUBJECTS
Objective: In the first-in-man clinical study of intermittent intraputamenal monthly infusions of CDNF in subjects with moderately advanced PD, we explored cerebrospinal fluid (CSF) biomarkers…Disease stage and nigrostriatal integrity predict motor progression in individuals with early Parkinson’s disease
Objective: To identify predictors of motor progression in individuals with early Parkinson’s disease (PD), who are either untreated or treated with monoamine oxidase B (MAO-B)…
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