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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

Advancing Research for Neurological Diseases Act

Reps. Burgess (R-TX) and Van Hollen (D-MD) have introduced HR 292, the Advancing Research for Neurological Diseases Act of 2015. The bill would create a national data collection system at the Centers for Disease Control (CDC) focused on neurological diseases, …

Patient Focused Drug Development

Parent Project Muscular Dystrophy (PPMD) is taking the lead on strengthening the voice of the patient throughout the drug and larger medical product development process. They are doing this by urging congress to add  transparency requirements in upcoming 21st Century Cures …

The Coalition for Health Funding: Sign On to Stop Sequestration

Sequestration is upon us again as the temporary and partial relief that Chairman Ryan and Chairman Murray negotiated in the Bipartisan Budget Act expires at the end of FY 2015. With the very real threat of the return of these

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

Take action to support the OPEN ACT, HR 971 (Orphan Product Extensions Now, Accelerating Cures & Treatments), which helps remove drug development roadblocks and incentivizes the development of treatments for the benefit of all rare disease patients. Biopharmaceutical companies possess …

Alliance for a Stronger FDA

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