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…Metabolomic Analysis of Fecal Matter from Parkinsonian Mice
Objective: Assess the metabolic changes in fecal matter in mice treated with an immunomodulatory therapy. Background: The cause of neuronal death in Parkinson’s disease (PD)…K0706, A Potent Orally Bioavailable Brain-Penetrating Selective Inhibitor of cABL Protein Tyrosine Kinase, Exhibits Neuroprotective Activity in Preclinical Models of Parkinson’s Disease
Objective: Preclinical assessment of K0706, a potent c-Abl inhibitor, as a neuroprotective disease modifying agent for the treatment of Parkinson’s disease (PD) related neurodegeneration using…
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