Development of an AAV-mediated artificial miRNA targeting human alpha-synuclein (α-syn) as a potential gene therapy for Synucleinopathies
Objective: To develop an adeno-associated virus (AAV)-based artificial miRNA (miRNA) system targeting human α-syn. Background: Synucleinopathies such as Parkinson’s disease (PD) and Multiple System Atrophy…Correlation between specific lower limb muscle strength and postural instability and gait difficulties in Parkinson’s disease
Objective: To explore the relationship between the strength of specific lower limb musclegroups and their associations with PIGD motor features. Background: Postural instability and gait…Investigating the genetic relationship between vitamin B12 deficiency and Parkinson’s disease
Objective: The aim of this study was to investigate the genetic association and causal link between vitamin B12 and the risk and progression of Parkinson's…Small Molecule Inhibitors for Precise Inhibition of α-Synuclein Oligomer Generation in Parkinson’s Disease (PD)
Objective: To develop small molecule therapeutics for PD that inhibit production of toxic α-synuclein oligomers and reduce aggregate formation, targeting the molecular mechanisms at the…Rasagiline in Chinese patients with early-stage parkinson’s disease: a randomized, double-blind, placebo-controlled, delayed-start trial
Objective: Objective: This randomized, double-blind, placebo-controlled, delayed-start trial aimed to investigate the efficacy in symptom improvement and disease-modifying effect of rasagiline for Chinese patients with…Preliminary Report on the efficacy of Allogeneic Bone marrow-derived Mesenchymal Stem Cells as a disease-modifying therapy for idiopathic 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) delivered intravenously to slow the progression of…AAV5-miHTT gene therapy mediates sustained mutant huntingtin lowering in brain and cerebrospinal fluid of Huntington’s disease minipigs up to 5 years
Objective: Our objective is to assess long-term safety of and durability of an adeno-associated viral vector serotype 5 encoding an engineered miRNA (AAV5-miHTT) targeting human…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…
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