Rare Disease News

Rare Cancer Day 2020 To Take Place on September 30

Published September 16, 2020 by NORD

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Washington, DC, September 16, 2020–The National Organization for Rare Disorders (NORD®) has announced the second annual Rare Cancer Day, taking place September 30, 2020. Spearheaded by the NORD Rare Cancer Coalition®, which is composed of 27 rare cancer-specific member organizations, Rare Cancer Day is observed to bring …

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A Newborn Screening Reflection: Sarah’s Story

Published September 15, 2020 by NORD

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September is Newborn Screening Awareness Month, and I’d like to share our family’s experience with it, and why we think every parent should know more about this incredible test, that …

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Voices of Rare Cancer: Jeremy’s Story

Published September 11, 2020 by NORD

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In this week’s Voices of Rare Cancer feature, Jeremy shares his experience with pseudomyxoma peritonei (PMP), and the role genomic testing has played in his treatment.

2020 NORD Summit to Feature Leading Experts in Public Policy, Patient Advocacy, Rare Disease Research and Regulatory Science

Published September 10, 2020 by NORD

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Washington, DC, September 10, 2020–At a time when it is vitally important for the rare disease community to come together, the National Organization for Rare Disorders (NORD®) has announced a program of …

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September is Newborn Screening Awareness Month

Published September 1, 2020 by NORD

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A Personal Reflection from Stephanie Cozine, Delaware Rare Action Network Ambassador Did you know that your baby is screened for different diseases depending on which state you live in? That …

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Head of the Herd: Deb Ayres, President, The Avalon Foundation

Published August 28, 2020 by NORD

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At the Head of the Herd is… Deb Ayres, President, The Avalon Foundation. Deb advocates for… patients with pediatric hypophosphatasia (HPP) and their families. Deb is located in… Toledo, Ohio. How Deb got here… When my daughter Avalon (“Avie”) was ten …

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NORD CEO Affirms Science Should Guide FDA’s Work

Published August 25, 2020 by NORD

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“Since 1906, FDA’s dedicated career employees have worked tirelessly to ensure that Americans have access to the safest supply of products we rely on every day, including food, drugs and …

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NORD Releases Four Guiding Principles on Telehealth Issues

Published August 17, 2020 by NORD

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The COVID-19 pandemic has had a huge impact on the rare disease community. NORD has heard from countless patients over the last few months about how this crisis has added to the …

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Head of the Herd: Emile Najm, KAT6A Foundation

Published May 22, 2020 by NORD

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At the head of the herd is… Emile Najm, Chief Executive Officer, KAT6A Foundation Emile most frequently checks his… Facebook account Emile advocates for those living with… A rare syndrome …

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NORD to Host an Externally-led Patient-Focused Drug Development Virtual Meeting on Krabbe Disease, Thursday, October 29

Published May 13, 2020 by NORD

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NORD, KrabbeConnect and Partners for Krabbe Research invite you to register for an externally-led Patient-Focused Drug Development (EL-PFDD) virtual meeting on Krabbe disease, taking place Thursday, October 29, 12:30-4:30pm ET. …

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