Developing infrastructure for a multi-arm multi-stage trial in Parkinson’s
Objective: To scope and develop infrastructure for the first multi-arm multi-stage (MAMS) platform trial for Parkinson’s disease, the Edmond J Safra ACT-PD initiative (EJS ACT-PD).…Involving people with lived experience of Parkinson’s in trial design: the EJS ACT-PD initiative
Objective: To maximally involve people with the lived experience of Parkinson’s in the design of the first multi-arm multi-stage (MAMS) trial for disease modifying therapies…Exploring Young people’s role in providing relief to grandparents affected with Alzheimer
Objective: In some families, children never know that their parents or grandparents are affected by the Parkinson’s Disease (PD). Whatever be the way that Movement…Longitudinal brain perfusion changes correlated with UPDRS Total in a Phase I mesenchymal stem cell trial for Parkinson’s disease
Objective: Explore changes in correlations between brain perfusion and UPDRS-Total (UPDRS-T) after a single infusion of allogeneic bone marrow-derived mesenchymal stem cells (MSC). Background: Our…Clinical and biomarker characteristics of Huntington’s Disease patients recently proposed for huntingtin-lowering clinical trial inclusion
Objective: We investigated plasma NfL levels, as well as clinical measures, in Huntingtin (Htt)-mutation carriers 48 years of age and under, with a CAP score…Neuroprotective trial design methodology for Parkinson’s: a scoping review
Objective: To further understanding of the diversity and impact of trial design choices in trials of disease modifying therapy (DMT) in Parkinson’s disease (PD). Background:…Vodobatinib, a potent, orally bioavailable brain-penetrating inhibitor of c-Abl as a potential neuroprotective agent for the treatment of Parkinson’s Disease
Objective: To compare and contrast nilotinib and vodobatinib regarding efficacy, brain penetration, and safety. Background: Preclinical evidence suggests that c-Abl is critical for pathogenesis of…An AAV9 encoding human ABCD1 (SBT101) shows functional improvement following spinal cord delivery in a rodent model of adrenomyeloneuropathy
Objective: SBT101 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy candidate delivering a functional copy of the human ABCD1 gene that is in development…Nutritional Status of Patients with Parkinson’s Disease: Report from Indonesian Tertiary Referral Hospital
Objective: This study aimed to describe and determine the prevalence of nutritional status in outpatient Parkinson’s Disease (PD) patients. Background: Parkinson’s disease is the most…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…
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