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…Informing the design of disease modifying trials for Parkinson’s disease – A Delphi study
Objective: To assemble an international, multi-stakeholder Delphi panel to seek consensus on trial methodology for an adaptive trial platform investigating disease modifying therapies (DMTs) in…STATINS IN PARKINSON’S DISEASE: INFLUENCE ON MOTOR PROGRESSION
Objective: To evaluate whether long-term statin use may affect motor progression in a large cohort of de novo patients with Parkinson's Disease. Background: Statins are…Attitudes on therapy-associated risks in individuals with multiple system atrophy and Parkinson’s disease and their views on aspects of clinical trials
Objective: To assess risk attitudes of patients suffering from multiple system atrophy (MSA) and Parkinson’s disease (PD) on future disease-modifying therapies as well as related…
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