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Web Desc
From Rare to Care: Discovery, Modeling and Translation of Rare Diseases
Organizer(s): Josef M. Penninger and Kym Boycott
Date: November 11 - 14, 2018
Location: Vienna BioCenter, IMP Lecture Hall, Vienna, Austria
Developed in collaboration with Vienna BioCenter Research Institutes GMI, IMBA,
IMP and MFPL

Sponsored by Bioverativ Therapeutics, Moderna, Novo Nordisk A/S, Sanofi US, Sarepta Therapeutics and Valerion Therapeutics
Summary of Meeting:
Work over the past 30 years has resulted in the identification of genes for ~50% of the estimated 7,000 rare genetic diseases; it is predicted that most of the remaining disease genes will be identified in the next 10 years. Approximately 500 medicinal products are currently on the market for rare diseases. The accelerating pace of rare disease gene identification means, in effect, an almost commensurate increase in molecularly defined, readily diagnosable, but nonetheless poorly understood and untreatable diseases. This conference will examine the current and future bottlenecks to gene discovery, disease modeling and therapeutic approaches and suggest strategies to enable progress in this regard. Ultimately, successful deployment of precision medicine for rare diseases will inform such approaches more broadly.
Scholarship Deadline: July 25 2018
Discounted Abstract Deadline: July 25 2018
Abstract Deadline: August 13 2018
Discounted Registration Deadline: September 18 2018
Keystone Symposia thanks our Sponsor(s) for generously supporting this meeting:
Bioverativ TherapeuticsModernaNovo Nordisk A/SSanofi USSarepta TherapeuticsDeveloped in collaboration with VBC-Institutions and Vienna BioCenter
We gratefully acknowledge additional support from these exhibitors at this conference:
BioLegend, Inc.HITECH Projekt-Handels & Service GmbHLEXOGENThermo Fisher Scientific
We gratefully acknowledge additional support for this conference from:
Bio-Rad LaboratoriesTecniplast Deutschland GmbHValerion Therapeutics
We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:

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

National Center for Advancing Translational Sciences (NCATS)
Grant No. 1R13TR002524-01
Funding for this conference was made possible (in part) by 1R13TR002524-01 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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