Rare Disease News

In Final Installment of Video Series, Patient Advocacy Group Leaders Share Thoughts on the Importance of Participation in RDCA-DAP

Published December 16, 2020 by NORD

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The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) initiative will allow patient organizations to leverage the data they have collected to accelerate understanding of disease progression, clinical outcome measures and biomarkers …

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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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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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NORD and Coalition Representing Millions of Patients Urge Policymakers to Adopt “Patient-First” Policy Agenda

Published November 3, 2020 by NORD

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NORD joined a coalition of 33 other patient organizations representing millions of people with pre-existing conditions to release our top priorities for a new administration. “The 100 Days Agenda: A …

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NORD and Patient Advocacy Groups Host an EL-PFDD Meeting on Krabbe Disease

Published November 2, 2020 by NORD

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[carousel id=”48517″] One of the most important gatherings of the Krabbe disease community occurred in collaboration with the US Food and Drug Administration (FDA) on October 29, and it was 100% virtual with live, interactive components. FDA regulators, academic …

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NORD Announces RareLaunch Workshops to Support Those Seeking to Start Rare Disease Nonprofits or Research Programs

Published October 28, 2020 by NORD

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Washington, DC, October 28, 2020—As part of its commitment to community engagement and organizational capacity building, the National Organization for Rare Disorders’ (NORD®) RareLaunch® training program will host two days of free workshops to take place in December, funded in part through a grant …

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