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:…Molecular Integration in Neurological Diagnosis (MIND): A Center-Wide Approach to Improve Molecular and Genetic Characterization of Parkinson’s Disease
Objective: To develop a clinical and genetic repository of all patients with Parkinson’s disease at The University of Pennsylvania Parkinson’s Disease and Movement Disorders Center…Using cognitive computing to identify existing drugs with potential to stabilize mitophagy pathways associated with Parkinson’s Disease
Objective: We applied IBM Watson for Drug Discovery (WDD), a cognitive computing platform that uses natural language processing, to identify compounds with the potential to…The mGlu2/3 orthosteric antagonist LY-341,495 abolishes the anti-dyskinetic and anti-psychotic effects of the mGlu2 positive allosteric modulator LY-487,379 in the MPTP-lesioned marmoset
Objective: We seek to determine the effect of orthosteric blockade of metabotropic glutamate 2 and 3 (mGlu2/3) receptors with LY-341,495 on the anti-dyskinetic and anti-psychotic…LY-341,495, an mGlu2/3 antagonist, abolishes the anti-dyskinetic and anti-psychotic effects of the mGlu2/3 orthosteric agonist LY-354,740 in the MPTP-lesioned marmoset
Objective: We aim to determine the effect of orthosteric blockade of metabotropic glutamate 2 and 3 (mGlu2/3) receptors with LY-341,495 on the anti-dyskinetic and anti-psychotic…TFEB-driven expression prevents neurodegeneration in a multiple system atrophy mouse model
Objective: To explore the potential neuroprotective effects of overexpressing the transcription factor EB in a MSA mouse model . Background: Synucleinopathies are neurodegenerative diseases characterized…Longitudinal analysis of the modified Hoehn and Yahr disease stage in PD-1101, a Phase 1b clinical study of VY-AADC01
Objective: To describe changes over the first year in disease severity and activities of daily living using modified Hoehn and Yahr (mH&Y) stage and UPDRS…Investigation of the protective efficacy of alpha-synuclein-specific antibodies in a novel neuronal cell model for synucleinopathies
Objective: To investigate the protective efficacy of different alpha-synuclein (α-Syn)-targeting antibodies in a new model of α-Syn cell-to-cell spread. Background: The 14 kDa protein α-Syn…Translating Preclinical Data to a Human Equivalent Dose for AMT-130 AAV Gene Therapy for Early Manifest Huntington’s Disease
Objective: To extrapolate the human equivalent dose (HED) for a Phase I/II clinical trial in adults to explore the safety, tolerability, and efficacy of striatally-administered…The SUNRISE-PD Study, a clinical trial of AXO-LENTI-PD: a CNS-directed gene therapy for the treatment of Parkinson’s Disease
Objective: This presentation will provide an overview of the design and key objectives of the SUNRISE-PD study. Background: Parkinson’s disease (PD) is caused in part,…
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