Keystone Symposia is very pleased to hold our second rare disease conference and our first conference in a series of annual conferences to be held in beautiful Vienna, Austria, developed in collaboration with Vienna BioCenter Research Institutes GMI, IMBA, IMP and MFPL. 

Work over the past 30 years has resulted in the identification of genes for approximately 50% of the estimated 7,000 rare genetic diseases, and 500 medicinal products are currently on the market for rare diseases. It is predicted that most of the remaining disease genes will be identified in the next 10 years. 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. The three-day conference will:

• Examine the current and future bottlenecks to gene discovery, disease modeling and therapeutic approaches for the estimated 7,000 known rare diseases;
• Explore platforms for enabling global cooperation and data sharing to address the grand challenges of rare diseases;
• Bring together diverse stakeholders (clinicians, basic scientists, industry, government, patient advocates), as well as students and post-doctoral fellows, to forge cross-disciplinary, international collaborations to advance rare disease research and therapy.

The conference will feature the following confirmed invited speakers (additional short talk speakers will be added, chosen from abstracts):