Wall Street Journal Features an Article on the Importance of Rare Disease Research

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.

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2014 Rare Disease Legislative Conference Presentations

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.

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SCAD Alliance at Rare Disease week 2014

“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

Read more about SCAD Alliance at Rare Disease Week in Washington D.C. 2014

Rare Disease Week in D.C. featured in Florida Newspaper

“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

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]