Read About the Legislation Featured at Lobby Day

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.

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Feb. 27th Rare Disease Congressional Caucus Briefing

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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

Panelists:

  • 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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Briefing Sponsors:

logos for breifing

Rare Disease Day Cocktail Reception & Documentary Screening

Please join RDLA for our 4th Annual Rare Disease Cocktail Reception & Screening of the HBO Documentary Film Life According to Sam to kick-off Rare Disease Day Week in Washington, DC.

Tuesday, February 25, 2014
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Rare Disease Legislative Conference & Lobby Day 2014

Join RDLA and 200 rare disease advocates in Washington, DC for Rare Disease Day – Week, February 25th – 27th 2014. 

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Join us in Washington DC: Rare Disease Day – Week Feb. 25th – 28th

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.

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2013 Rare Disease Legislative Conference Presentations

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  1. Welcome – Overview of the Day

    Julia Jenkins, Rare Disease Legislative Advocates

  2. Message from Abbey Meyers on the Passage of the Orphan Drug Act
  3. The Power of One: Advocacy Matters

    Anthony J. Castaldo, US Hereditary Angioedema Association

  4. Back to Basics: HIV/AIDS Advocacy as a Model for Catalyzing Change

    Margaret Anderson, FasterCures

  5. The Creating Hope Act: An Advocacy Success Story

    Mobilizing advocates and working with Congressional Staff to introduce & pass legislation

  6. CureTheProcess: Improving the FDA Regulatory Process

    Building a grassroots coalition to move a policy discussion to successful legislation

    Emil Kakkis, MD, PhD, EveryLife Foundation for Rare Diseases

  7. Rare Disease Congressional Caucus

    • Helen Dwight, Office of Rep. Lance (R-NJ)
    • Nicole Cohen, Office of Rep. Crowley (D-NY)
  8. Overview of the Political & Legislative Process

    Make up of 113th Congress

    Jen Bernstein, JC White Consulting

  9. Health Funding: The Budget and Appropriations Process

    Updates on FDA, NIH funding and the threat of Sequestration

  10. Health Reform Overview

    State Based Exchanges and Essential Health Benefits

    Joel White, Council for Affordable Health Coverage

  11. Engaging Government Agencies

    Working to expand newborn screening

    Jim Bialick, Newborn Coalition

  12. Rare Disease Awareness: Genes Ribbons on the Hill

    Nicole Boice, Global Genes Project

Thank You to our Event Sponsors:

Conference Sponsors [/raw]

RDLA’s 3rd Annual Rare Disease Cocktail Reception & Movie Screening

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

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Rare Disease Day at NIH (RDD@NIH)

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.

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My Experience on Capitol Hill: Rare Disease Lobby Day

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.

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Rare Disease Week: The Fighting Four Musketeers

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.

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