Rare Disease News

The Undiagnosed: Orphans of the Rare Disease Community

Published October 5, 2015 by NORD

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While the approximately 7,000 known rare diseases run the full spectrum of symptoms and experience, rare disease patients encounter many common obstacles. Rare patients face incredible difficulty finding safe and …

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NORD Issues Statement Applauding the Approval of Ensuring Access to Clinical Trials Act

Published September 29, 2015 by NORD

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Advocacy

The following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), on yesterday’s approval of the Ensuring Access to Clinical Trials …

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American Plasma Users Coalition (A-PLUS) Applauds Recent Move by Department of Health and Human Services to Limit Out-of-Pocket Costs

Published September 21, 2015 by NORD

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As part of the American Plasma Users Coalition (A-PLUS), NORD and several of its member organizations submitted letters to Department of Health and Human Services Secretary Sylvia Mathews Burwell and …

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NORD Issues Statement on FDA Rejection of Citizen Petition

Published September 17, 2015 by NORD

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Advocacy

On September 10, the Food and Drug Administration (FDA) notified the National Organization for Rare Disorders (NORD) that it was rejecting NORD’s Citizen Petition submitted in 2011 to request that …

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Rare Disease Day 2016 Theme and Slogan Announced!

Published September 16, 2015 by NORD

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Theme: Patient Voice Slogan: Join us in making the voice of rare diseases heard It’s here! As the official sponsor and host of Rare Disease Day in the U.S., NORD …

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Rare Disease Day 2016 Theme and Slogan Announced

Published September 16, 2015 by NORD

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Theme: Patient Voice Slogan: Join us in making the voice of rare diseases heard As the official sponsor and host of Rare Disease Day in the U.S., NORD is excited …

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Bennet, Burr, Warren, Hatch Introduce Bill to Help Patients by Facilitating Faster Development of Drugs for Rare Diseases

Published September 15, 2015 by NORD

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Advocacy

Today, the Senate introduced the Advancing Targeted Drugs for Rare Diseases Act of 2015 (see press release below). According to NORD President and CEO Peter L. Saltonstall, “NORD supports legislation that …

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FDA: Another tool helping developers navigate the difficult road to approval of drugs for rare diseases

Published September 15, 2015 by NORD

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Today, the FDA released a blog post about a new tool for drug developers. The FDA writes, “With the vast majority of rare diseases still without FDA-approved treatments, we have …

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Urge Your U.S. Senators to Attend Sept. 16th Briefing on Neurological Disease Research

Published September 14, 2015 by NORD

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Advocacy

Invite your U.S. Senators to attend a briefing on the Advancing Research for Neurological Diseases Act (S. 849) on September 16 from 11:30 to 12:30 in the Senate Russell Building, …

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NORD’S Free Student Membership: Sign up Today!

Published September 2, 2015 by NORD

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A few months ago, NORD and our members hosted a “Meet the Patients” event at the annual conference of the American Medical Student Association (AMSA). We were thrilled with the …

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