Are we underestimating the dysautonomic burden in GBA related a-synucleinopathies?
Objective: The aim of the present study is to understand the prevalence of dysautonomic symptoms in a cohort of Italian glucocerebrosidase (GBA) mutated patients with…Clinical Management of Incipient Wearing-off with the 19-Items Wearing off Questionnaire (WOQ-19)
Objective: To evaluate the use of the 19-items Wearing off Questionnaire (WOQ-19) to detect fluctuations in patients with non-advanced Parkinson's disease (PD) and to evaluate…Can we hear pathology in premanifest Huntington’s disease? Acoustic and listener-based analysis of speech
Objective: The present study evaluated the ability of acoustic and listener-based perceptual measures to differentiate speech deficits in the premanifest and early stages of HD…The Roche HD Natural History Study – an external comparator by design
Objective: The Roche Natural History (NH) study in Huntington’s disease (HD) (NCT03664804) is a prospective, longitudinal cohort study designed to provide further clinical validation of…Burden of Huntington’s disease in the US: Analysis of an insured population
Objective: To describe the healthcare resource utilization (HRU) in Huntington’s disease (HD) in private (commercial) and public (Medicare and Medicaid) insurance plans in the US.…Sustained mutant huntingtin lowering in the brain and cerebrospinal fluid of Huntington disease minipigs mediated by AAV5-miHTT gene therapy
Objective: To assess the translatability and long-term efficacy of gene therapy-mediated huntingtin (HTT)-lowering in a large animal model of Huntington disease (HD), transgenic HD (tgHD)…A randomized clinical trial of multimodal balance training with rhythmical cues: effects on freezing of gait in Parkinson’s disease
Objective: To investigate whether the multimodal training is more effective in improving balance and gait in freezers versus non-freezers. Background: Non-pharmacological interventions are increasingly used…Lack of Notable Skin Reactions from a Novel Levodopa/Carbidopa Prodrug after 10 Days of Repeated 24-Hour Continuous Subcutaneous Infusion at the Same Administration Site
Objective: To assess the skin safety and tolerability of 24-hour continuous subcutaneous infusion (CSCI) of ABBV-951. Background: Therapeutic approaches for advanced Parkinson’s disease (aPD) patients…Effect of an Increase in Dose of Istradefylline, an A2A Receptor Antagonist, in Levodopa (LD)-Treated Patients with Parkinson’s Disease (PD)
Objective: To observe whether increasing the istradefylline dose from 20 to 40mg/day provides more patients with meaningful clinical responses. Background: Istradefylline, a well-tolerated selective adenosine…Design, characterization and In vivo evaluation of intranasal delivery of levodopa loaded microspere for Parkinsonism
Objective: Levodopa is the drug of choice in the treatment of Parkinson's disease but it exhibits low oral bioavailability and very low brain uptake. The…
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