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Web Desc
Rare and Undiagnosed Diseases: Discovery and Models of Precision Therapy
Organizer(s): William A. Gahl and Christoph Klein
Date: March 05 - 08, 2017
Location: Fairmont Copley Plaza, Boston, MA, USA
Sponsored by Moderna Therapeutics and Vertex Pharmaceuticals Incorporated
Summary of Meeting:
Of the 7000 known rare diseases, only a fraction have their molecular and mechanistic bases delineated, and many other diseases have yet to be discovered. Characterization of these disorders will reveal new biochemical pathways and cell biological processes, point to potential drug targets, and provide hope for millions of affected individuals. This meeting will address approaches and best practices in pursuing rare and undiagnosed disorders, limitations in data sharing as a barrier to new disease discovery, and examples of successful descriptions of new diseases, mechanisms, and treatments. Specific aims include fostering the development of expertise in rare diseases and the definition of their natural histories; an international network of scientists and physicians collaborating on new disease discovery by sharing phenotypic and sequence data; and new therapeutic approaches based upon rare and new disease mechanisms. Through this meeting, participants should become familiar with rare and undiagnosed disease programs, acquire insights into new disease mechanisms, learn about potential therapeutic targets, and establish collaborations that enhance rare disorder expertise and new disease discovery. The meeting will bring together physicians who are expert in rare disorders with scientists who know metabolic pathways and mechanisms, advancing understanding and therapy. A desired outcome of the meeting includes developing lists of clinical and basic research experts for synergistic collaborations, and creating an international organization to perpetuate the joint investigation of rare and new diseases.
Scholarship Deadline: November 2 2016
Discounted Abstract Deadline: November 2 2016
Abstract Deadline: December 6 2016
Discounted Registration Deadline: January 10 2017
Keystone Symposia thanks our Sponsor(s) for generously supporting this meeting:
Moderna TherapeuticsVertex Pharmaceuticals Incorporated
We gratefully acknowledge additional support for this conference from:
EveryLife Foundation for Rare Diseases
Wilhelm Foundation
We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:

RaNA Therapeutics Inc.

Roche
We gratefully acknowledge the generous grant for this conference provided by:

National Center for Advancing Translational Sciences (NCATS)
Grant No. 1R13TR001915-01
Funding for this conference was made possible (in part) by Grant Number TR 001915 - 01 from the National Institutes of Health. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention of trade names, commercial practices, or organizations imply endorsement by the U.S. Government.
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Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:

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