From Rare to Care: Discovery, Modeling and Translation of Rare Diseases

Organizer(s): Josef M. Penninger and Kym Boycott

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

For important information on the coronavirus, please click here

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

Abstract submission is now closed. Registered attendees may bring a poster onsite. Please contact our office at +1 800-253-0685; +1 970-262-1230 or email info@keystonesymposia.org if you are interested.

Keystone Symposia thanks our Sponsor(s) for generously supporting this meeting:

We gratefully acknowledge additional support from these exhibitors at this conference:

We gratefully acknowledge additional support for this conference from:

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 by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.

We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:

Click here to view more of these organizations

Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:

Click here to view more of these organizations