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Multi-pronged Approach to Reposition Drugs for Parkinson’s Disease
December 4, 2012
… Drug Repositioning Congress - Washington, DC Isradipine. Pioglitazone. Droxidopa. What do these drugs have in common? They are all approved for other indications and are … to reduce cost, mitigate risk and accelerate time to get the treatment to the market. This talk will describe …
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Targets to Therapies: Neuroinflammation Target Validation
… Objective: The Targets to Therapies Program seeks to build a robust evidence … potential relevance to Parkinson’s disease. Funding will support projects to clarify human relevance, define …
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Targets to Therapies: Endolysosomal Target Validation
… Objective: The Targets to Therapies Program seeks to build a robust evidence … potential relevance to Parkinson’s disease. Funding will support projects to clarify human relevance, define …
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Mutations in GBA Gene Linked to Parkinson's Cognitive Decline
December 1, 2016
… MJFF support. As we learn more about genetic connections to Parkinson's disease (PD), researchers strive to … What does having a GBA mutation really mean for someone? Complicating matters further is that there are many different mutations on one gene. How does the type of mutation on that gene impact PD? ( …
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AAV-Based Approach for Regulatable and Cell Type-Specific Gene Therapy in PD: Restoring Excitability to an Intrinsically Silenced Subpopulation of GP Neurons
… disrupts the GPe–STN synchronous activity, elevates PD motor symptoms; this provides a direct evidence for … We propose to develop gene therapy based on the use of recombinant adeno-associated virus (rAAV) vectors with … gene therapy in PD patients. This work led to additional support from MJFF to continue these studies. …
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Rapid and Ultra-sensitive Quantitation of Disease-associated and Synuclein Seeds in Brain and Cerebrospinal Fluid by Syn RT-QuIC
… clusters of the protein a-synuclein (αSyn) that appears to cause the disease. Two such tests have been reported by … that our new Parkinson’s test, called αSyn RT-QuIC, will show high diagnostic accuracy for PD, while being much faster … and controls, and allowing levels of abnormal αSyn to be compared in treated and untreated patients over the course …