The Second Conference on Clinical Research for Rare Diseases

The Rare Diseases Clinical Research Network and the Clinical and Translational Science Awards (CTSA) program along with other partners are pleased to announce a one-day conference in Bethesda, Md., focused on conducting clinical research in rare diseases. Speakers include expert clinical investigators who represent a spectrum of medical diseases and disciplines and who are actively engaged in rare disease research, as well as members of the NIH and industry. The conference will culminate with a dinner keynote address by John Crowley, whose story was featured in the film Extraordinary Measures. Crowley is the former CEO of Novazyme Pharmaceuticals, Inc., and president and CEO of Amicus Therapeutics, Inc.

The full-day program will include a series of short lectures with ample time for questions plus panel discussions by experts in rare diseases research. Topics will include:
• Study design and biostatistics for small sample sizes
• Recruitment strategies for rare diseases
• Using the resources of the CTSA program
• Pathways for developing orphan products (FDA)
• Working with patient advocacy groups
• Working with industry on rare disease research
• Career development in rare diseases research

New investigators, trainees, junior faculty and others interested in rare diseases research methodology will have the opportunity to present their work through submission of an abstract, which may be selected for the poster session; investigators working on rare diseases will provide feedback and advice. The registration fee is $100 and includes all meals, refreshments and meeting materials. Travel awards are available on a competitive basis. To learn more, visit rarediseasesnetwork.epi.usf.edu/conference/.

Contact Information
RDCRNconference@epi.usf.edu