Rare Disease News

National Spotlight on FDA and Duchenne Muscular Dystrophy: What This Means for Everyone with a Rare Disease

Published November 20, 2015 by NORD

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Next week, a Food and Drug Administration panel of experts will hold the first of two meetings to review two drugs designed to slow the muscle wasting effects associated with Duchenne …

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NORD Publishes Guide to Promote Physician Awareness of Rare Lung Disorder

Published October 28, 2015 by NORD

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Medical

NORD Publishes Free Online Guides to Encourage Diagnosis and Treatment for Patients The National Organization for Rare Disorders (NORD) has published a Physician Guide to Nontuberculous Mycobacterial Lung Disease to …

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Raise Your Voice: Rare Disease Day 2016 is Coming

Published October 12, 2015 by NORD

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Date: February 29, 2016 Global Theme: Patient Voice Global Slogan: Join us in making the voice of rare diseases heard By definition, each rare disease affects a small segment of …

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Rare Disease of the Day

Published September 24, 2015 by NORD

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Von Hippel-Lindau disease (VHL)

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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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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