Rare Disease News

Historic “Voice of the Patient” Report on Pyruvate Kinase Deficiency Now Available on FDA Site

Published April 28, 2020 by NORD

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“…As we pull into the hospital, she starts screaming that she doesn’t want to be there, and she wants to be home… No matter how much her doctors want to …

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NORD Joins 400+ Groups to Prevent Discrimination During COVID-19 Triaging

Published April 24, 2020 by NORD

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Between 25 and 30 million Americans have a rare disease, many of whom are disproportionately at risk of getting sick from COVID-19. According to the Centers for Disease Control and …

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Update on NORD’s Recent Advocacy Work on COVID-19 for the Rare Community

Published April 20, 2020 by NORD

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In this time of crisis and uncertainty, NORD’s policy team has been working to pursue policies at the federal and state levels to address COVID-19 related concerns critical to the …

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Rare Disease Day 2020 Advocacy Events Recap

Published March 13, 2020 by NORD

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In 2009, NORD was invited to join the official Rare Disease Day campaign, led internationally by EURODIS. Since then, NORD has been the official US sponsor of Rare Disease Day, …

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Rare Disease Day 2020 and the Mission I Choose to Accept, by Debbie Drell, Director of Membership for NORD

Published March 11, 2020 by NORD

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If you or someone you love is diagnosed with a rare disease, you might find yourself on a very quick learning curve about the disease (or about the lack of …

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Head of the Herd: Akiva Zablocki, The Hyper IgM Foundation

Published March 6, 2020 by NORD

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At the head of the herd is… Akiva Zablocki, President, The Hyper IgM Foundation Akiva most frequently checks his… https://www.facebook.com/Akiva Akiva advocates for those living with… hyper IgM syndrome Akiva …

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NORD Announces Sing Me a Story Raffle for Upcoming Living Rare, Living Stronger Patient and Family Forum

Published March 3, 2020 by NORD

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NORD is proud to welcome Sing Me a Story to the upcoming 2020 Living Rare, Living Stronger Forum in Cleveland, Ohio, where the ideas of children living with rare diseases will be turned into song! This opportunity …

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Savannah’s Story in honor of Rare Disease Day

Published February 29, 2020 by NORD

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I suffer from neuromyelitis optica, or NMO disease (sister of MS, also known as Devic syndrome) and chronic pain. I experienced my first attack in the summer and now am …

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Bridget’s Story in honor of Rare Disease Day

Published February 29, 2020 by NORD

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I was diagnosed with afibrinogenemia when I was just one day old. The day I was born my heel sticks would not stop bleeding, which immediately had the delivering doctor …

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Ashlee’s Story in honor of Rare Disease Day

Published February 29, 2020 by NORD

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I was diagnosed with primary immunodeficiency when I was four years old. With primary immunodeficiency, the assumption is that “it’s just an infection” or that it’s normal because “kids get …

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