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…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…Fingolimod-responsive complex hyperkinetic movement disorder in neuromyelitis optica spectrum disorder
Objective: Highlight a complex hyperkinetic movement disorder in Neuromyelitis optica spectrum disorder exacerbation with remission on fingolimod therapy. Background: Neuromyelitis optica spectrum disorder (NMOSD) can…Treatment Selection in Multi-Arm Multi-Stage Clinical Trials in Parkinson Disease: The Search for the Ideal Neuroprotective Drug
Objective: The Edmond J Safra Accelerating Clinical Trials in Parkinson disease (PD) initiative (EJS ACT-PD) aims to accelerate the identification of disease modifying treatments for…Selection of clinical doses for SBT101, an AAV9-hABCD1 vector for the treatment of adrenomyeloneuropathy
Objective: To inform selection of doses of SBT101 for a first-in-human clinical study. SBT101 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy candidate intended…
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