The 201 Trial in Untreated Parkinson’s Disease
Objective: To evalute the safety, tolerability, clinical benefit and protein pathology status following risvodetinib (IkT-148009) treatment in participants with untreated Parkinson's disease. Background: RIsvodetinib is…the role of the “unfolded protein response” and the perk pathway in parkinson’s disease: study of genetic polymorphisms
Objective: To study a possible association between polymorphisms of the genes coding for proteins involved in the UPR and the development of PD or PD…GRoningen Early-PD Ambroxol Treatment (GREAT) Trial: A randomized, double-blind, placebo-controlled, single center trial with ambroxol in Parkinson patients with a GBA-mutation
Objective: To study the potential disease-modifying properties of ambroxol in PD patients with a GBA1 mutation Background: The most common genetic risk factor for Parkinson’s…DPPX Autoimmune Encephalitis Associated with Severe Multifocal Dystonia.
Objective: To describe a novel motor phenomenology in an individual diagnosed with anti-DPPX encephalitis at our institution and to compare it with previously known phenotypic…Rationale and Design of the Slowing Parkinson’s Early through Exercise Dosage – United Kingdom (Slow-SPEED-UK) Trial
Objective: Slow-SPEED-UK is a randomized controlled trial investigating the effects of an app-based exercise program on individuals at higher risk of Parkinson’s Disease (PD). Background:…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.…
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