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…Innovating Clinical Trial Design to Accelerate Assessment of Disease-Modifying Treatments for Parkinson’s: The EJS ACT-PD Initiative
Objective: To design an optimal multi-arm, multi-stage (MAMS) platform trial for disease-modifying therapies (DMTs) in Parkinson’s disease (PD). Background: The current clinical trials process cannot…Development of therapeutic substances for the disassembly of α-synuclein aggregates as disease-modifying treatment of synucleinopathies
Objective: Development of high-affinity ligands to α-synuclein (α-syn) for a therapeutic approach aiming for the disassembly of α-syn aggregates as a disease-modifying treatment of synucleinopathies.…Design of a time-to-event study in GBA Parkinson’s Disease patients: Efficacy and safety of BIA 28-6156, an allosteric activator of beta-glucocerobridase (GCase)
Objective: To assess the efficacy of BIA 28-6156 in delaying clinically meaningful motor progression in PD patients who have a PD-risk associated variant in the…Preliminary Report on the Safety and Tolerability of Allogeneic Bone marrow-derived Mesenchymal Stem Cells as a disease-modifying therapy for Parkinson’s disease: Phase IIa double-blind, randomized controlled trial
Objective: To select the safest and most effective number of repeat doses of allogeneic mesenchymal stem cells (MSC) purified from bone marrow-derived from a healthy…Anle138b-P1-02: A randomised, double-blinded, placebo-controlled phase 1b study to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of the oligomer modulator anle138b in Parkinson´s disease
Objective: To present first-in-patient data of the oligomer modulator anle138b (TEV-56286, emrusolmin) in patients with Parkinson’s disease (PD). Background: Synucleinopathies such as PD and Multiple…Protective potential of laughter yoga and clapping exercise on stress level and motor functions in Parkinson’s disease patients with type 2 diabetes
Objective: To examine the protective potential of laughter yoga and clapping exercise on blood glucose regulation on stress level and motor functions of PD patients…Neuroprotective properties of arylpiperazine-sulphonamides in an in vitro model of Parkinson’s disease
Objective: To investigate the neuroprotective effects of novel arylpiperazine compounds as measured by viability of SH-SY5Y cells in vitro. Background: Parkinson’s disease (PD) is a…
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