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…Clinical Manifestations of Metabolic Syndrome in Parkinson’s Disease – Report from Indonesia Tertiary Referral Hospital
Objective: The study aims to determine the prevalence of metabolic syndrome and related factors in Parkinson's disease (PD). Background: Parkinson's disease is a neurodegenerative disorder,…Sex differences in vascular risk factors for Parkinson’s disease
Objective: Our aim is to illustrate the incidence of vascular risk factors (VRF) in a cohort of patients with confirmed Parkinson's disease (PD) and characterize…GT-02287, a brain-penetrant structurally targeted allosteric regulator for glucocerebrosidase show evidence of pharmacological efficacy in conduritol β-epoxide (CBE) models of Parkinson’s disease
Objective: Demonstrate the involvement of our structurally targeted allosteric regulator Parkinson’s disease (PD) candidate, GT-02287, in decreasing the CBE-induced neurotoxic effect and in restoring lysosomal…Designing a multi-arm multi-stage trial for Parkinson’s
Objective: The Edmond J Safra ACT-PD initiative (EJS ACT-PD) is developing the first multi-arm multi-stage (MAMS) platform trial for potential disease modifying approaches for Parkinson’s…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…Homeostatic Improvement of Brain Bioenergetic Metabolism in Parkinson’s Disease: Results From A Phase 2 REPAIR-PD Clinical Trial With CNM-Au8
Objective: To determine the effects of the nanocatalytic drug, CNM-Au8, on brain energy metabolites in Parkinson’s Disease. Background: Converging lines of evidence are identifying brain…
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