The March 21st edition of the Wall Street Journal features an article by noted health journalist Amy Marcus titled “Hard Choices in Pursuit of Rare-Disease Cures: Patients Urge More Data Gathering as Tighter Federal Budget Crimps Research” which details the importance of rare disease research and the struggles many groups face as budget cuts hamper their pursuit of a cure.
RDLA’s 2014 Legislative Conference took place on Feb. 25 at the National Press Club in Washington D.C. Over 160 rare disease advocates were in attendance, with 25 speakers educating and training the advocates how to make their voices heard. Click on the title to view the video from the conference.
“As anticipated, Rare Disease Day 2014 events did not disappoint. We are taking the information we gained and are busy reaching out to new contacts to advance the mission of SCAD Alliance.” – SCAD Alliance
“Cape woman fights for rare-illness sufferers, honors her son with advocacy.”
A few weeks ago, Hickman, who now lives in Cape Coral, went once again to Washington, D.C., to speak to politicians for the Rare Disease Legislative Conference & Lobby Day. Read more
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.