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  • Share-Your-Rare-Stories

    Share Your Rare Story

    During Rare Disease Week in D.C. on Wednesday the 25th & Thursday the 26th your “Rare Story” will be hand delivered by advocates to the offices of your Members of Congress.

    By sharing your story, representatives can get a glimpse …

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  • Congressional-Report-Cards

    2014 RDLA’s Legislative Scorecard

    Rare Disease Legislative Advocates has compiled a congressional scorecard to educate advocates on how their Representative has voted on issues affecting the rare disease community.  Each Representative’s score is based on membership in the Rare Disease Congressional Caucus as well …

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    Rare Disease Week 2015 On Capitol Hill

    Registration For

    Legislative Conference

    Opens January 5th, 2015!

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  • 21st century cures

    House Energy & Commerce 21st Century Cures Initiatives

    From A summary by M. Nielsen Hobbs, “The Pink Sheet: Biomedical Reform Legislation Draft Could Be Relesed by January”

    At the final roundtable for the 21st Century Cures initiative in Washington, Chairman Upton promises swift action, and urges stakeholders to

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Overview of Legislation Benefiting the Rare Disease Community

Passed in the 113th Congress:

  • Gabriella Miller Kids First Research Act: Redirects $12.6 Million in funding from political conventions to the National Institutes of Health (NIH) Common Fund to support transformative research for childhood cancer, autism, Fragile X syndrome and other rare pediatric diseases. With the odds stacked against us, more than 70 organizations called on Congress and ensured this essential funding was in the 2015 omnibus appropriations bill.

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RareVoice Nominee, Julie Flygare featured in article about awards

From: Sleep Review by Associate Editor, Cassandra Perez

Julie Flygare, JD, founder of the non-profit organization Project Sleep and the Narcolepsy: Not Alone campaign, was recently recognized at the RareVoice Awards Gala in Washington, DC, hosted by Rare Disease Legislative Advocates. The gala is designed to honor advocates who have made a difference in the fight against rare diseases, according to the EveryLife Foundation for Rare Diseases. Julia Jenkins, executive director of the EveryLife Foundation, explains that the public submits nominations to the group for these awards to recognize outstanding work in this area.

The event was emceed by news anchor Greta Kreuz and actor Jonny Lee Miller, who was also honored with an Abbey for speaking on Capitol Hill to urge Congress to spur the development of treatments for rare diseases. Representative Doris Matsui (D-CA) was honored… to read entire article go to:


Senators Hatch (R-UT) & Bennet (D-CO) Introduce Dormant Therapies Act

From the Office of Senator Hatch:

Washington, D.C.—Senators Orrin Hatch, R-Utah and Michael Bennet, D-Colorado, introduced the Dormant Therapies Act, a bill that will establish a new class of pharmaceuticals known as “dormant therapies” eligible for 15 years of data protection.  This provision will remove the “ticking patent clock” conundrum that forces companies to  prioritize research based on which compounds can be brought quickly to market.

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Take Action

Call Your Senators To Urge Them To Vote YES on the ABLE Act TODAY!

Today is the last day congress is likely to be in session. Your voice matters. The ABLE Act would ease the financial burden for individuals with disabilities by creating tax-free savings accounts.

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Update: Newborn Screening Saves Lives Re-Authorization Act Has Passed and is Off to the President to be Signed Into Law

March of Dimes is urging the House to swiftly vote on H.R. 1281, the Newborn Screening Saves Lives Re-authorization Act as amended and passed by the Senate.  This bipartisan legislation will help ensure that infants continue to receive comprehensive, effective, …

Sign-on to Support the OPEN ACT: Encourage Companies to Repurpose Drugs for Rare Diseases!

Take action to support the OPEN Act (HR 5750), which helps remove drug development roadblocks and incentivizes the development of novel treatments for the benefit of all rare disease patients. Biopharmaceutical companies possess a wealth of scientific knowledge from drugs …

Action Needed: Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care

Rare Disease Patients and their families will be dramatically impacted by the Draft Guidance on Disclosing Reasonably Foreseeable Risks in Research Evaluating Standards of Care recently released for comment by the Office of Human Research Protections. We are writing to …
Alliance for a Stronger FDA

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