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CASK GENE RESEARCH STRATEGY

CASK GENE RESEARCH STRATEGY

Project CASK believes a cure for CASK-related disorders is possible. Getting there requires close, transparent and iterative collaboration among basic and translational researchers, clinicians, biotechs, pharmaceutical companies and the CASK community.

 

It also requires an engaged community working with urgency to contribute to the toolkit of research assets necessary for testing therapeutic options and to make strategic investments to move the science as quickly and safely as possible toward improving the lives of people with CASK gene disorders and their families.

RESEARCHERS

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CASK THINK TANK

One of the first initiatives of Project CASK will be a CASK Think Tank, virtually bringing together 25-35 scientists from across disciplines and institutions to work collaboratively to identify the most critical unanswered questions about CASK gene disorders and the most promising strategies to a cure. The findings from this meeting will inform the elaboration of the Roadmap to a Cure as well as the first request for research proposals. 

 

A continually focused version of the Think Tank will be held on an annual basis to take stock of CASK research, update the Roadmap, and prioritize the key avenues of inquiry for the next year to ensure that all actions are directly contributing toward advancing treatments and a cure. Some of the Think Tank participants will form the CASK Science Collaborative.

CASK SCIENCE COLLABORATIVE

The CASK Science Collaborative is the first of its kind for CASK-related disorders and will convene scientists from diverse disciplines and approaches to exchange perspectives and incremental discoveries of relevance to accelerate the path to a cure. 

 

The Collaborative will enable Project CASK to harness the speed of discoveries and new technologies for curing rare diseases and ensure an efficient and transparent research strategy. This collective effort will limit duplication of research, end laboratory isolation, and promote continuous learning and knowledge-sharing. 

 

The CASK Science Collaborative will have twice annual meetings as well as organize ad hoc research updates with Project CASK grantees to ensure new findings can systematically inform ongoing research, prospective research and the overall research strategy.

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