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Mar. 24, 2014

TOPIC: Advocacy, Featured News

Making Sure The Rare Disease Patient Voice is Heard on Capitol Hill

Posted by Mary Dunkle

From Left to Right: Stephanie Bozarth, Andrew Emmett, Sandy Robinson, Peter L. Saltonstall, Richard Zyne, Charles Mohan

NORD recently hosted a Capitol Hill briefing on the topic “Rare Diseases in a Changing Healthcare Landscape”. Keeping our elected officials informed and aware is a critically important, and this briefing was very well received.

View a video of this event.

We thank our speakers, who did a fantastic job:

  • Stephanie Bozarth of the National MPS Society
  • Charles Mohan of the United Mitochondrial Disease Foundation
  • Richard Zyne of CurePSP
  • Sandy Robinson of Avalere Health
  • Andrew Emmett of the Biotechnology Industry Organization (BIO)

Stephanie, Chuck and Richard talked about current issues of importance from the patient perspective, including research funding, Medicare and Social Security, the need for treatments and diagnosis delay.

Sandy talked about patient access to therapies and the impact of the Affordable Care Act on the rare disease community.

Andrew discussed recent advances  and the need to continue to improve the ways in which new drugs are developed and reviewed to bring safe, effective therapies to patients who currently don’t have any.

We would like to thank all who participated for helping us make sure the rare disease patient voice is heard on Capitol Hill!

3 Responses to “Making Sure The Rare Disease Patient Voice is Heard on Capitol Hill”

  1. Margee' Morris says:

    I wish someone would make Capitol Hill recognize all of the Nam Vets that are dying of Glioblastoma Multiforme Stage 4 Brain Cancer. It cannot be a coincidence that all of these Nam Vets are dying 40+ years later of the same disease.

  2. I wrote many comments on the Rare Disease Blog page.

    I am happy to report that others with my rare disease Parry Romberg

    Syndrome that I helped advocate through my own story has helped others like me also obtain

    SSD insurance.

    Other patients must come forward and advocate

    in behalf of their rare disease and have the courage to

    tell and share their own stories on many websites because

    so many people don’t even know or heard of most rare diseases.

    Find the courage and tell your stories!

  3. Ann Hallett says:

    I have stiffperson syndrome and would like to see more research in this area. I also have an unknown disorder that causes severe vomiting and I need to go to the hospital frequently for IV meds. I wish she I got in a crisis I could be treated at home but I can my afford the medications. Do you know that I picked a box of 12 suppositions it cost me 117

    00. I can’t afford that