Rare Disease News

Patient Advocacy Group Leaders Tell How Data Sharing Can Help with Understanding Rare Diseases in New Video

Published December 9, 2020 by NORD

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Natural history studies provide health care professionals and researchers with first-hand information about people living with rare diseases and give insight into how rare diseases present, progress over time and impact patients. Data sharing supports patients by helping …

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New Video Series Featuring Patient Advocacy Group Leaders, “The Importance of Patient-Reported Data to Progress in Rare Disease,” Launched by NORD, C-Path

Published December 3, 2020 by NORD

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As the primary benefactors of drugs that treat rare diseases, patients and caregivers must play a role in the drug development process. Patients and caregivers bring a human perspective to the drug development process …

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NORD and Partners Publish Abstract Based on Web-Based Hemophilia A Educational Initiative for Patients, Caregivers and HCPs

Published November 24, 2020 by NORD

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The National Organization for Rare Disorders (NORD®), in partnership with PlatformQ Health, the Hemophilia Federation of America and the National Hemophilia Foundation, is pleased to announce the publication of an …

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Connecticut Magazine Features NORD in Article on CT Nonprofits

Published November 20, 2020 by NORD

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On 11.19.20 in an article entitled “Ways to Make Your Donated Money and Goods Go Further,” Connecticut magazine included NORD in their list of ten Connecticut-based nonprofits that have each …

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NORD Releases Report on the State of Rare Disease Diagnosis, Care and Treatment in America Over Three Decades

Published November 19, 2020 by NORD

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Washington, DC—November 19, 2020: Today, the National Organization for Rare Disorders (NORD®) published a comprehensive report through its RareInsights™ initiative: Barriers to Rare Disease Diagnosis, Care, and Treatment in the US: A 30-year Comparative Analysis. As part of an ongoing mission to generate evidence …

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NORD Launches Project RDAC, Announces Inaugural Meeting on December 16

Published November 18, 2020 by NORD

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Washington, DC, November 18, 2020—With a mission of empowering and equipping the rare disease community to effectively engage decision-makers within their state governments, the National Organization for Rare Disorders (NORD®) has launched an initiative that will assist in …

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Telehealth Report from NORD Covered in Managed Healthcare Executive

Published November 13, 2020 by NORD

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NORD’s recent telehealth report was featured in an article in Managed Healthcare Executive, a digital magazine from MJH Life Sciences, on 11.12.2020: “The federal government took important steps to temporarily …

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Head of the Herd: Charlene Son Rigby, President and Cofounder, STXBP1 Foundation

Published November 13, 2020 by NORD

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At the Head of the Herd is… Charlene Son Rigby, President and Cofounder, STXBP1 Foundation Charlene advocates for those affected by… STXBP1 encephalopathy Charlene is located in… San Francisco, CA …

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NORD & C-Path Present 3-Part Video Series on Challenges of Rare Disease Drug Development, How RDCA-DAP Can Help

Published November 11, 2020 by NORD

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There are five steps in the drug development process, which are designed to help ensure that potential new therapies are both safe and effective. Developing medical products for rare diseases encounters …

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NORD Drives Rare Disease Progress Through Patient-Powered Data With IAMRARE™ Registry Members

Published November 10, 2020 by NORD

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Washington, DC, November 10, 2020—This year, in conjunction with its rare disease community partners, the National Organization for Rare Disorders (NORD®) continued to drive research and innovation through the IAMRARE™ registry program. Having launched its first study in 2014, today IAMRARE supports longitudinal data collection …

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