The NINDS Approach to Therapeutic Development for Ultra-Rare Neurological Disorders, Bridging Translational and Clinical Frontiers in Gene-based Therapy
Objective: To establish an innovative process for fast and efficient pathways of delivering gene-based therapy for ultra-rare neurological disorders from the lab to the clinic…Boosting Clinical Trial Power in Parkinson’s Disease with AI-Generated Digital Twins
Objective: To evaluate the incorporation of prognostic digital twins to enhance traditional clinical trials in Parkinson’s Disease (PD) for disease modifying therapies. We assess the…Muscle Augmentation Paradox: Contrasting Therapeutic Approaches in Myasthenia Gravis and Parkinson’s Disease
Objective: This exploration aims to dissect the therapeutic paradox presented by muscle augmentation strategies in Myasthenia Gravis (MG) and Parkinson's Disease (PD), offering a comparative…Lithium Therapy Associated with Reductions in Serum Neurofilament Light Chain (NfL) in Parkinson’s Disease.
Objective: To assess the effects of lithium aspartate and lithium carbonate therapy for 24 weeks on serum NfL in Parkinson’s disease (PD). Background: Lithium has…First-In-Human Single and Multiple Ascending Dose Trial Design of LY3962681, a Novel Intrathecally Delivered siRNA Targeting α-synuclein mRNA for the Treatment of Patients with Parkinson’s Disease
Objective: Development of a Phase 1 clinical trial evaluating LY3962681, a novel, intrathecally delivered siRNA directly targeting α-synucleinmRNA (SNCA) to reduce α-synuclein protein (ASYN), for the treatment of…Slow-SPEED-NL: study protocol for a remote trial of exercise for Parkinson’s disease prevention
Objective: To determine the feasibility and preliminary efficacy of a remote exercise intervention in prodromal Parkinson’s disease (PD). Background: Disease-slowing interventions have been ineffective in…TrkB modulator OT-003 reduces dopaminergic cell loss, mitochondrial stress, alpha-synuclein aggregation and motor dysfunction in Parkinson’s disease models.
Objective: To assess efficacy and potency of our proprietary lead compound OT-003, an orally bioavailable small molecule Tropomyosin receptor kinase B (TrkB) modulator, in preclinical…Exidavnemab offers opportunities as a potential disease modifying therapy in several synucleinopathies
Objective: Immunotherapy against α-synuclein is a promising novel treatment strategy for Parkinson's disease and related α-synucleinopathies. We have developed exidavnemab (BAN0805), a monoclonal antibody with…Recruitment of Ethnoracially Diverse Participants for Disease-Modifying Drug Trials in Parkinson’s Disease: Are We Doing It Right? A Systematic Review and Meta-Analysis.
Objective: To perform a systematic review and meta-analysis on recruitment of people with Parkinson’s disease (PwP, PD) from different ethnic and racial groups in disease-modifying…Chronic Sleep Fragmentation Accelerates the Onset of Prodromal Symptoms and Pathology in Parkinson’s Disease Model Mice
Objective: To investigate whether chronic sleep fragmentation accelerates the onset of prodromal symptoms and pathology of Parkinson’s disease (PD) in mice. Background: The prodromal phase…
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