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We are excited to announce this very special and inspiring Rare Disease Congressional Caucus Briefing

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


February 27th, 2014
12:00-1:30 PM
Capital Visitors Center, Room HVC-201
Lunch Provided by event sponsors Genzyme & Shire.
There is very limited space available, you MUST register if you would like to attend.
To Register Click this link:

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, CEO, Jonah’s Just Begun to Cure Sanfilippo
  • Jonny Lee Miller, Actor, Patient Advocate


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