MRI, Clinical, and Neuropathological Findings after Bilateral Intra-striatal Administration of rAAV5-miHTT in Non-human Primates
Objective: To assess the effects of intra-striatal administration of a huntingtin protein (HTT) lowering gene therapy in macaca fascicularis non-human primates (NHP). Background: The aggregation…Magnetic Resonance Spectroscopy Evaluation of Neuronal Integrity and Astrocytosis in a Phase 2 Study of Laquinimod as a Treatment for Huntington Disease (LEGATO-HD)
Objective: Evaluate the effect of laquinimod on putaminal and frontal white matter markers of neuronal integrity and astrocytosis using magnetic resonance spectroscopy (MRS) in patients…Brain MRI Volume Changes after 12 months laquinimod treatment of Huntington disease (LEGATO-HD)
Objective: To explore the effect of laquinimod on brain volume in patients with Huntington Disease (HD). Background: Volume loss in caudate and other brain regions…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…
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