RDCRN Web Site | Open Studies

 

 


 

 

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Background and Goals

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Program Agenda
Speaker Roster


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Travel Awards
Abstract Submission and Poster Requirements

Contact Information


RDCRN Web Site

Conference on Clinical Research for Rare Diseases

The 2nd Conference on Clinical Research for Rare Diseases will be held on Tuesday, September 21st, 2010

Program

See full agenda >>

The full day program included 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 with
    small sample sizes
  2. Recruitment strategies for rare diseases
  3. Availability of core translational research
    resources
  4. Maintaining anonymity for participants with rare
    diseases
  5. Use of demographic and registry data
  6. Pathways for developing orphan products (FDA)
  7. Working with patient advocacy groups
  8. Working with industry on rare disease research
  9. Career development in rare diseases research

 

 


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

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.

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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.


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Abstract and Poster Session

Conference attendees are encouraged to submit an abstract for review by the planning committee. Abstracts selected by the planning committee are invited to be displayed as a poster during the conference.

Investigators working on rare diseases provide feedback and advice during the poster session.

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