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…Funding and Sustainability Challenges 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…Longitudinal Exercise is Associated with Reduced Hospitalization Risk in Parkinson’s Disease
Objective: To determine if longitudinal exercise and physical therapy (PT) use is associated with reduced risk of hospital encounter in individuals with Parkinson’s disease (iwPD).…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…
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