Pridopidine for the Treatment of Early Huntington’s Disease
Objective: Assess the efficacy of pridopidine 45 mg bid for maintenance of total functional capacity (TFC) and motor function in early HD patients. Background: Pridopidine…Translatable biomarkers in gene therapy for Huntington’s Disease: learnings from pre-clinical studies
Objective: We have developed AAV5-miHTT, a recombinant AAV-based gene therapy expressing an engineered microRNA that specifically binds to HTT exon1, resulting in lowering of both…Increasing chances of detecting a neuroprotective signal in PhI/II PD clinical trials?
Objective: Design an early CT program to detect a neuroprotective signal in PD. Background: Attempts to develop PD modifying therapies have been largely disappointing so…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…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…
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