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)…LRRK2 Inhibition by BIIB122 / DNL151: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Ph 1 Healthy Volunteer and Ph 1B Parkinson’s Disease Trials
Objective: To evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of the LRRK2 inhibitor, DNL151 (BIIB122), in healthy volunteers (HVs) and Parkinson’s disease (PD) patients. Background:…Cinpanemab in Early Parkinson’s Disease: Phase 2 SPARK Study Results
Objective: To evaluate cinpanemab (BIIB054) efficacy and safety. Background: Aggregated alpha-synuclein (α-syn), a major constituent of Lewy bodies, is thought to play a central role…Parkinson Progression Marker Initiative – New Science/New Cohorts
Objective: The Parkinson Progression Marker Initiative (PPMI) is expanding its cohorts and exploring additional biomarkers of PD progression to test hypotheses of the underlying molecular…Cardiovascular Fitness, Plasma Biomarkers and Dopaminergic Denervation in Parkinson’s Disease
Objective: To examine the relationship between cardiovascular (CV) fitness, plasma biomarkers associated with PD progression and/or exercise and dopaminergic denervation, measured by I-123-Ioflupane single photon…PASADENA: A Phase 2 study to evaluate the safety and efficacy of prasinezumab in early Parkinson’s disease; Part 1 Week-52 results
Objective: Evaluate efficacy and safety of prasinezumab following 52 weeks of treatment in participants with early Parkinson’s disease (PD). Background: Prasinezumab is a humanized monoclonal…Using a Delphi process to inform the design of disease modifying trials in Parkinson’s disease
Objective: To consolidate current opinion on trial methodology of disease modifying therapies (DMTs) for Parkinson’s disease (PD) and establish consensus on a core protocol for…
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