Exploring dopamine transporter single-photon emission computed tomography quantification as a measure of disease progression in idiopathic Parkinson’s disease
Objective: To evaluate the ability of DAT SPECT quantification to track disease progression in subjects with idiopathic Parkinson's disease (PD) by comparing baseline scans to…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 single nigral injection of human ENGRAILED-1 induces long-lasting behavior benefit in an experimental primate model of Parkinson Disease
Objective: Evaluate the efficacy of a single nigral injection of human ENGRAILED-1 (hEN1) in a recognized chronic MPTP macaque model. Background: EN1 homeoprotein has been…Substantia Nigra (SN) Hyperechogenicity as seen in Transcranial Sonography (TCS) is a reliable disease progression marker from Stage I to Stage II in Early Onset Parkinson’s Disease (EOPD)
Objective: To test the hypothesis that SN hyperechogenicity seen in transcranial sonography (TCS) can be used as a reliable disease progression marker from stage I…Evaluation of neuroprotection by human ENGRAILED-1 in a chronic AAV-hα-synuclein-A53T (AAV-A53T) rat model of Parkinson Disease
Objective: Investigate the long-term neuroprotective effect of a single bilateral injection of human ENGRAILED1 (hEN1) in a rat α-synuclein model of Parkinson Disease (PD). Background:…Safety of Bone marrow-derived Allogeneic Mesenchymal Stem Cells in Parkinson’s disease Patients
Objective: Prove safety and tolerability of allogeneic mesenchymal stem cells (MSC) purified from bone marrow derived from a healthy adult and delivered intravenously in escalated…Pre-diagnosis physical activity habits are associated with age of diagnosis and postural instability in Parkinson’s disease
Objective: To determine if pre-diagnosis exercise and brain-derived neurotrophic factor (BDNF) genotype are predictive of age at diagnosis and severity of balance impairment. Background: Studies…
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