Rare Disease Congressional Caucus Briefing with Special Celebrity Guest

Rare Disease Legislative Advocates, National Organization for Rare Disorders & Jonah’s Just Begun-Foundation to Cure Sanfilippo, in coordination with Rare Disease Congressional Caucus Co-Chairs Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing.

Special Guest Jonny Lee Miller, who stars as Sherlock Holmes in the hit show Elementary, will help us unlock the mystery of the “Science Behind Rare Disease Policy”

Date and Location:
Thursday February 27, 2014
12:00 noon – 1:30 pm EST
Capitol Visitors Center, Room: HVC – 201

Lunch Provided by event sponsors Genzyme & Shire

Moderator:  Kay Holcombe, Senior Policy Analyst, Genzyme


  • P.J. Brooks, Ph.D., Health Science Administrator – Office of Rare Diseases Research & Division of Metabolism and Health Effects, NIAAA,  NIH
  • Marshall Summar, MD, NORD Board Member, Division Chief, Genetics and Metabolism at Children’s National Medical Center and Faculty, Children’s Research Institute, Center for Genetic Medicine Research
  • Emil Kakkis, MD, PhD, President EveryLife Foundation for Rare Diseases
  • Hugh Hempel, Founder, Addi & Cassi Fund
  • Jill Wood, CFO, Jonah’s Just Begun-Foundation to Cure Sanfilippo, Inc
  • Jonny Lee Miller, Actor, Patient Advocate

Limited Seating, You must RSVP to attend:



For more information, contact Jenny Carey, CHI’s Director of Federal Government Relations & Alliance Development (, or Julia Jenkins, Executive Director at Rare Disease Legislative Advocates (RDLA) (