January 21, 2003

NIH Muscular Dystrophy Research Task Force Agenda

One Democracy Plaza 
6701 Democracy Blvd., Suite 800
Bethesda, MD 20817
TEL: 301-594-5055

“Promoting Treatment”

January 21, 2003
7:00 - 9:00 p.m.

7:00 PM Welcome and Introductions
7:30 PM Meeting Goals
8:00 PM Review of Thursday Agenda
8:30 PM Discussion of Goals and Topics
9:00 PM Adjourn

January 22, 2003
8:30 a.m. - 3:00 p.m.

8:30 AM Review Agenda  
8:45 AM Treatments for Muscular Dystrophy  
      Symptoms being treated Introduction: Dr. Craig M. McDonald
      Target Tissues and Organs Introduction: Dr. Elizabeth McNally
      Outcome goals Introduction: Dr. Charles A. Thornton
      Outcome measures Introduction: Dr. Craig M. McDonald
10:15 AM NIH Office of Rare Diseases - Promoting Treatment
    Dr. Stephen Groft
10:45 AM FDA Office of Orphan Products Development - Orphan Drug Development
    Dr. John McCormick
11:15 AM MDA - Activities Promoting Treatment Development
    Dr. Sharon Hesterlee
    Dr. Rodney Howell
11:45 AM NIH Translation Research Initiatives
    Dr. Robert Baughman
12:00 PM Lunch  
12:15 PM Survey of Potential Therapies Introduction: Dr. Louis Kunkel
12:45 PM Identifying Priority Areas
Recruitment Strategy for Study Participants
1:30 PM Promoting cooperation by NIH, Federal agencies, health voluntary groups, and industry
2:30 PM Summarize  
3:00 PM Adjourn  
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