The 2nd Conference on Clinical Research for Rare Diseases took place on Tuesday, September 21st, 2010
Bethesda North Marriott, Bethesda, MD

Featured Speaker:

Keynote address was given by John Crowley, Former CEO of Novazyme Pharmaceuticals, Inc.; President & CEO of Amicus Therapeutics, Inc.

Other speakers included expert Clinical Investigators who represent a spectrum of medical diseases and disciplines and are actively engaged in rare disease research, as well as members of the NIH and Industry.

Program

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The full day program includes a series of short lectures with ample time for questions and discussion, plus panel discussions by expert researchers in rare diseases. 

Topics:

1. Study design and biostatistics for dealing withsmall sample sizes
2. Recruitment strategies for rare diseases
3. Utilizing the resources of the CTSA program
4. Pathways for developing orphan products (FDA)
5. Working with patient advocacy groups
6. Working with industry on rare diseases research
7. Career development in rare diseases research
   

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Attendees

Target audience includes new investigators, trainees, junior faculty, and others interested in rare disease research methodology.

Attendees 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.