During Rare Disease Week 2014 in Washington D.C. over 160 rare disease advocates lobbied for 3 main pieces of legislation and asked their legislators to join the Rare Disease Legislative Caucus.
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),
hosted 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”
Thursday February 27, 2014
12:00 noon – 1:30 pm
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, CEO, Jonah’s Just Begun to Cure Sanfilippo
- Jonny Lee Miller, Actor, Patient Advocate
Join RDLA and 200 rare disease advocates in Washington, DC for Rare Disease Day – Week, February 25th – 27th 2014.
You are invited to join RDLA and 200 rare disease advocates in Washington, DC for Rare Disease Day (Week), February 25 – 28, 2013. Below is an overview of the events.
Julia Jenkins, Rare Disease Legislative Advocates
- Message from Abbey Meyers on the Passage of the Orphan Drug Act
The Power of One: Advocacy Matters
Anthony J. Castaldo, US Hereditary Angioedema Association
Margaret Anderson, FasterCures
The Creating Hope Act: An Advocacy Success Story
Mobilizing advocates and working with Congressional Staff to introduce & pass legislation
Building a grassroots coalition to move a policy discussion to successful legislation
Emil Kakkis, MD, PhD, EveryLife Foundation for Rare Diseases
- Helen Dwight, Office of Rep. Lance (R-NJ)
- Nicole Cohen, Office of Rep. Crowley (D-NY)
Make up of 113th Congress
Jen Bernstein, JC White Consulting
Health Funding: The Budget and Appropriations Process
Updates on FDA, NIH funding and the threat of Sequestration
State Based Exchanges and Essential Health Benefits
Joel White, Council for Affordable Health Coverage
Working to expand newborn screening
Jim Bialick, Newborn Coalition
Nicole Boice, Global Genes Project
Thank You to our Event Sponsors:
Join RDLA for our 3rd Annual Rare Disease Cocktail Reception & Movie Screening to kick-off Rare Disease Day (week) in Washington DC
Monday, February 25, 2013
5:00pm Cocktail Reception * 6:30 Movie Screening * 8:00 Panel Discussion
Carnegie Institution of Science, 1530 P Street Northwest, Washington DC
On February 28, and March 1, 2013, the National Institutes of Health (NIH) will celebrate the sixth annual Rare Disease Day with a 2-day-long celebration and recognition of the various rare diseases research activities supported by the Office of Rare Diseases Research – National Center for Advancing Translational Sciences (NCATS), the NIH Clinical Center, other NIH Institutes and Centers; the Food and Drug Administration’s Office of Orphan Product Development; other Federal Government agencies; the National Organization for Rare Disorders; and the Genetic Alliance.
By Laura Welch
I am a 33-year-old mother of a 2-year-old boy and wife to a wonderful US Soldier. In July 2011 we moved from Seattle to DC. Hello sunshine!
On August 18, 2011, I was diagnosed with Epitheliod Sarcoma (ES), a rare and aggressive form of cancer. I did what anyone in this day and age would do; I consulted the bible that is Google! The prognosis was bleak, to put it mildly. However, I am of the mindset that if this disease is so rare that it has little data available, I will make my own data and try not to dwell on the figures in front of me.
By Parent Advocate Heather Earley
pictured above with Congressman Dold
What a whirlwind! The last week of February is already but a mere blink of an eye. My in-box and to-do lists, however, are the tell-tale signs that I was incredibly busy.