New Op-Ed With Details on 21st Century Cures Initiative

by Rep. Fred Upton,U.S. Congressman representing Michigan’s 6th District &  Rep. Diana DeGette, Chief Deputy Whip

Originally published by The Huffington Post on May 6th:  Talk to any American adolescent and it quickly becomes clear that keeping pace with 21st century technology is a constant challenge. But staying on top of today’s technology goes far beyond trending hashtags and Instagram likes; it presents a unique opportunity to bring researchers, innovators, caregivers, and patients together in a new way that advances our collective understanding of disease and how we combat it.

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Rare Disease Advocate featured in CNN article about Compassionate Use

The program that allows patients to use experimental drugs not yet approved by the FDA is called “compassionate use”. Sandy Barker wonders, as do many others, if there might be a way to make it a little more compassionate.    

The CNN article gives a detailed overview of the compassionate use approval use process, including how it can be improved. It also illustrates how drug companies are increasingly concerned about how patients and parents of patients are using social media as a tactic in gaining approval for compassionate use.

RDLA’s April Advocate Luncheon/Conference Call

Advocate’s Luncheon/Conference Call

Wednesday, April 23rd, 2014 12 noon to 1:30 pm

Location: Offices of Polsinelli 1401 Eye Street NW 8th floor Washington, DC,  20005

Girl, 7, with Rare Disease Stands Beside Congressman as He pushes for More Funding

Stephanie stood beside Congressman Mike Fitzpatrick when he called for continued research funding for Tuberous Sclerosis Complex (TSC).

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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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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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Why FDA Supports a Flexible Approach to Drug Development

FDA Voice, Margaret A. Hamburg M.D., February 6, 2014

We all know that just as every person is different, so too is every disease and every drug. And so we weren’t surprised by the results of a new study published in the Journal of the American Medical Association. The study found that FDA used a range of clinical trial evidence when approving 188 novel therapeutic drugs for 208 indications (uses) between 2005 and 2012. These results are entirely consistent with our regulatory mandate. We believe varying approaches to clinical studies to support drug approval is good news, not bad. Read more…

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:

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