The Grassroots Movement Behind ULTRA/FAST & How you can join the Movement!

By Julia Jenkins, EveryLife Foundation Director of Government Relations

We are very excited to share the news that rare disease Accelerated Approval language made it into the House draft PDUFA bill. The language that incorporates the goals of ULTRA is the only mention of rare diseases in the entire 205-page bill. This is a huge accomplishment for the rare disease community and is a major step toward spurring the development of lifesaving treatments. While there is still work to be done, and it is too soon to pop open the champagne, I will take a few moments to tell the story of how a few parent activists ignited the grassroots patient community to influence the legislative process.

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Rare Disease Week: The Fighting Four Musketeers

By Parent Advocate Heather Earley
pictured above with Congressman Dold

What a whirlwind! The last week of February is already but a mere blink of an eye. My in-box and to-do lists, however, are the tell-tale signs that I was incredibly busy.

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The Origin of RDLA by Emil Kakkis, MD PhD

RDLA, the Rare Disease Legislative Advocates: the story behind an association developed to help all rare disease groups succeed

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Turning Science into Medicine – a Political Perspective

By Julia Jenkins, Director of Public & Government Relations

Our Government has an immense capability to help advance treatments and cures for rare diseases through incentives for industry, research grants, and improving regulatory conditions; however, our politicians often lack the public support to challenge the status quo and make good changes to the system.

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