The conduct of clinical research in rare diseases is essential.
In order to assure the future of rare diseases research, the training of the next generation of investigators in this field is important.
Who should attend?
New investigators, trainees, junior faculty, and others interested in rare disease research methodology. Attendees will have the opportunity to:
- Meet key leaders in rare diseases research.
- Engage in formal and informal networking and idea generation with other trainees and investigators.
- Discuss career development and strategic planning for academic success.
- Explore research collaborations.
Carol McAlear, MA
View past conference resources
Background & Goals
There are more than 6,8001 diseases classified as "rare" (defined as having a prevalence in the United States of <200,000 persons). While individually these entities are uncommon, as a group they are an important cause of chronic illness, disability and premature death in both children and adults. They take a disproportionate share of our health care dollars because of their complexity and the expense of, in most cases, inadequate therapy.
Despite their rarity, many fundamental advances in medicine have come from the study of rare diseases and these have benefited common diseases. As an example, the study of the human genome began with identification of rare diseases (muscular dystrophy being the first) and epigenetics was first identified in rare disorders. Both because of currently inadequate therapy and the potential to assist common as well as rare disorders, the conduct of clinical research in rare diseases is essential.
In order to assure the future of this research, the training of the next generation of investigators in this field is important. We are the ideal group to sponsor a conference addressing rare disease research methodology that would supplement general training in clinical research and help attract trainees and junior faculty into this important field. Learn more about us.