A double-blind randomized controlled trial of cognitive training for freezing of gait in Parkinson’s disease
Objective: To investigate the potential of cognitive training to improve symptoms of freezing of gait in Parkinson's disease. Background: Freezing of gait (FOG) is a…Development of a human neuroblastoma model of pantothenate kinase-associated neurodegeneration
Objective: To establish a stable human neuronal model defective in PanK2. Background: Pantothenate Kinase-Associated Neurodegeneration (PKAN), is caused by mutations in PANK2, which encodes one…A healthy volunteer phase 1 study of RE-024, a potential phosphopantothenate replacement therapy for patients with pantothenate kinase-associated neurodegeneration (PKAN)
Objective: To assess the safety, tolerability, and pharmacokinetics of single oral doses of RE-024 in healthy adult volunteers. Background: PKAN is a rare, autosomal recessive,…Significant improvement of adult-onset dystonia with cannabinoids-based oromucosal spray
Objective: We performed an open study of delta-9-tetrahydrocannabinol and cannabidiol (THC:CBD) oromucosal spray (Nabiximols, Sativex®) in 6 patients with suboptimal responsive adult-onset dystonia. Background: Cannabinoids…RE-024: A potential phosphopantothenate replacement therapy in 2 patients with pantothenate kinase-associated neurodegeneration (PKAN)
Objective: To evaluate the safety and potential efficacy of RE-024 in open treatment of 2 patients with PKAN. Background: PKAN is an autosomal recessive, neurodegenerative…Are Parkinson’s disease futility studies futile?
Objective: Revisit Phase II futility studies. Background: Published results of a Phase III trial of creatine monohydrate for Parkinson's disease (PD) in the presence of…Strategies to improve drug development efficiency through regulatory interactions, minimizing the placebo effect and improving data integrity within a phase 3 study of tozadenant in Parkinson’s disease patients with motor fluctuations (TOZ-PD)
Objective: To identify and implement strategies to improve the efficiency of drug development in Phase 3 studies. Background: Conducting global clinical trials can be challenging,…Risk factors for premature withdraw from the LS-1 PD study
Objective: To identify risk factors for premature withdrawal from a large, multi-center Parkinson's disease clinical trial of possible neuroprotection. Background: Premature withdrawal (PW) of subjects…Effects of subthalamic deep brain stimulation in motor and nonmotor function in Parkinson’s disease patients with parkin mutation
Objective: The aim of our study was to evaluate prospectively the changes in motor, nonmotor symptoms (NMS) and quality of life produced by subthalamic deep…Deep brain stimulation effect on the quality of life of individuals with Parkinson’s disease
Objective: To identify the perception of QOL in individuals with PD pre and post DBS. Background: The DBS is one type of effective treatment in…