Quantitative Motor (Q-Motor) Assessments Suggest a Beneficial Central Effect of Laquinimod in a Phase II Study in Huntington Disease (LEGATO-HD)
Objective: Evaluate the change from baseline at week 52 in Q-Motor (Quantitative Motor) measures, exploratory, standardized, and rater-independent outcomes in the LEGATO-HD study. Background: LEGATO-HD…Sustained mutant huntingtin lowering in the brain and cerebrospinal fluid of Huntington disease minipigs mediated by AAV5-miHTT gene therapy
Objective: To assess the translatability and long-term efficacy of gene therapy-mediated huntingtin (HTT)-lowering in a large animal model of Huntington disease (HD), transgenic HD (tgHD)…Path to Prevention (P2P) – Developing a Prodromal PD Progression Biomarker Program
Objective: The overall objective of the Path to Prevention (P2P) initiative is to identify clinical, imaging, and biologic markers of progression during the prodromal phase…A first-in-human clinical study to test the safety and preliminary efficacy of CDNF in Parkinson’s disease
Objective: To develop a novel disease-modifying therapy in Parkinson's disease (PD) that affects both motor and non-motor symptoms, based on a novel stress-protective protein CDNF.…Oral treatment with JNX3001 protects dopaminergic function in a non-human primate model of Parkinson’s disease alpha-synucleinopathy
Objective: We evaluated the efficacy of JNX3001 in an aSyn-based macaque model with plasma exposures associated with efficacy in rodents. Background: Alpha-synuclein (aSyn) deposition is…Motivators, Barriers and Clinical Correlates of Physical Activity in Parkinson Disease
Objective: To characterize physical activity levels as well as barriers and motivators for exercise in US military Veterans with Parkinson disease (PD). Background: Physical activity…Nurr1 gene: A new research target for Parkinson’s disease
Objective: The objective of our study is to determine if Nurr1 gene, a transcription factor essential for dopamine (DA) neuron development and survival, is a…T lymphocytes in Parkinson’s disease pathogenesis and therapy: A systematic review
Objective: A systematic literature review was conducted to synthesize the current state of knowledge on the role of T lymphocytes in Parkinson’s disease (PD) pathology…Activation of chaperone-mediated autophagy reduces oligomeric alpha-synuclein accumulation in LRRK2(R1441G) knockin mouse model of Parkinson’s disease (PD)
Objective: To determine age-dependent accumulation of oligomeric alpha-synuclein in the brain of aged LRRK2(R1441G) knockin mice; and to explore whether activation of chaperone-mediated autophagy (CMA)…Enhancing protein palmitoylation is protective in alpha-synuclein dependent cytotoxicity
Objective: To determine if enhancement of protein palmitoylation benefits alpha-synuclein (aS) dependent vesicular trafficking defects and cytotoxicity. Background: Recent studies suggest that vesicular trafficking defects…
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