Improving the design of clinical trials in early-stage Parkinson’s disease by using the estimand framework
Objective: To show how the estimand framework can be used to improve design of clinical trials testing treatments in early-stage Parkinson’s disease (PD). Background: The…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…
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