IkT-148009 as a potential disease-modifying therapy in PD
Objective: Modeling Parkinson’s disease in mice suggests c-Abl activation is required for PD initiation and progression, and therefore inhibition of c-Abl could be a strategy…Addressing pathological alpha-synuclein aggregation using a small molecule strategy
Objective: The discovery of cell-permeable, orally bioavailable and brain penetrant small molecules with the potential of inhibiting aggregation and intracellular accumulation of pathological alpha-synuclein (a-syn)…Fingolimod-responsive complex hyperkinetic movement disorder in neuromyelitis optica spectrum disorder
Objective: Highlight a complex hyperkinetic movement disorder in Neuromyelitis optica spectrum disorder exacerbation with remission on fingolimod therapy. Background: Neuromyelitis optica spectrum disorder (NMOSD) can…Treatment Selection in Multi-Arm Multi-Stage Clinical Trials in Parkinson Disease: The Search for the Ideal Neuroprotective Drug
Objective: The Edmond J Safra Accelerating Clinical Trials in Parkinson disease (PD) initiative (EJS ACT-PD) aims to accelerate the identification of disease modifying treatments for…Selection of clinical doses for SBT101, an AAV9-hABCD1 vector for the treatment of adrenomyeloneuropathy
Objective: To inform selection of doses of SBT101 for a first-in-human clinical study. SBT101 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy candidate intended…Challenges in Outcome Measure Selection in Multi-Arm Multi-Stage Clinical Trials in Parkinson Disease
Objective: The Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (PD) initiative (EJS ACT-PD) aims to accelerate the identification of disease modifying treatment for…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…
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