Rare Disease News

Raise Your Hand for Rare Disease Research

Published February 3, 2017 by NORD

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Click here to donate to research!

Orphan Drugs Represent 41 Percent of All New Medications

Published January 11, 2017 by NORD

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Washington, D.C., January 11, 2017—Data released by the U.S. Food and Drug Administration (FDA) show that 9 of the 22 new medications approved in 2016, or 41 percent, are orphan drugs to …

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Family Moves Across State Lines to Receive Better Access

Published December 22, 2016 by NORD

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Today we are sharing the story of one little girl who, by the age of three-months old, had faced surgery, an investigational treatment, and a near-fatal infection. When Gabby was born …

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A Message from Our President and CEO

Published December 20, 2016 by NORD

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“When you are faced with a difficult health challenge, you need to be able to turn to a trusted source for support and advice – much like many people battling …

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Reflections on FDA Listening Session

Published December 17, 2016 by NORD

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Advocacy

Earlier this week, I attended a listening session with the Commissioner of the FDA, Dr. Robert M. Califf. As a new employee in the NORD Policy Office, this was my …

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President Signs 21st Century Cures Medical Innovation Bill Into Law

Published December 13, 2016 by NORD

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Today, President Obama signed into law the 21st Century Cures Act, a game-changing bill for medical innovation. The bill includes many provisions that will improve the discovery, development, and delivery …

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State Report Card to Help People with Rare Diseases

Published December 13, 2016 by NORD

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Washington, D.C., December 13, 2016—A majority of states are not measuring up on legislative solutions that reduce the burden of rare diseases affecting 30 million Americans, according to a new …

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Expansion of Medical Foods Coverage for Military Families Heads to the President, Having Passed in the Senate

Published December 8, 2016 by NORD

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Advocacy

We are pleased to announce that today that the United States Senate has passed the National Defense Authorization Act (S.2943) with language substantially expanding coverage for medical foods in the …

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Senate Passes Landmark 21st Century Cures Act

Published December 7, 2016 by NORD

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The President is expected to sign the bill into law later this week.

House Votes to Expand Medical Foods Coverage for Military Families

Published December 2, 2016 by NORD

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Advocacy

We are pleased to announce that today the House of Representatives has passed the National Defense Authorization Act (S.2943) with language that substantially expands coverage for medical foods in the …

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