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NORD CEO Statement on the Termination of the Advisory Committee on Heritable Disorders in Newborns and Children

Published April 4, 2025 by NORD

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The sudden termination of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) raises serious concerns for the rare disease community and families nationwide. The ACHDNC is a critical part of the U.S. newborn screening system, providing guidance to the Secretary of the Department of Health and Human Services (HHS) on the most appropriate application of universal newborn screening tests, technologies, policies, guidelines, and standards....

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News & Articles

First Rare Disease Therapy Developed with a NORD IAMRARE Patient Registry Secures FDA Approval

Published June 5, 2025 by NORD

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QUINCY, Mass., June 5, 2025 – The National Organization for Rare Disorders (NORD®) proudly announces a major milestone: for the first time, patient data collected through the NORD IAMRARE® patient …

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Find Your Herd Through Running for Rare This Global Running Day

Published June 4, 2025 by NORD

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Running for Rare

Living with a rare disease means that Amanda is one of 30 million-plus “zebras” in the United States – and while she may be rare, she’s not alone. Amanda is …

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In Memory of Kate O’Bara: Raising Awareness for Vascular EDS

Published May 27, 2025 by NORD

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Patient Stories

By Laura O’Bara for Ehlers-Danlos Syndrome Awareness Month When you asked me to tell you how Ehlers-Danlos syndrome (EDS) type IV affected my daughter Kate’s life, I had to stop and think …

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FDA CBER Director Dr. Vinay Prasad to Keynote NORD’s Inaugural Rare Disease Scientific Symposium, Convening Top Researchers, Industry Leaders, and Federal Regulators

Published May 23, 2025 by NORD

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WASHINGTON, May 23, 2025 –The National Organization for Rare Disorders (NORD®) will convene leading voices in science, industry, and regulation at its inaugural Rare Disease Scientific Symposium, a two-day conference …

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In Honor of Eddie — A Story of Courage, Love, and an Unbreakable Bond

Published May 15, 2025 by NORD

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Patient Stories

For MPS Awareness Day, May 15 By Debra Bell, mother of Eddie James Bell Jr. (July 29, 1981 – April 4, 2022) It’s hard to know where to begin when …

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Montana Establishes Rare Disease Advisory Council to Support Residents Living with Rare Conditions

Published May 14, 2025 by NORD

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NORD’s advocacy helps Montana become 32nd state to create a dedicated council HELENA, MONT., May 14, 2025 — The National Organization for Rare Disorders (NORD®) celebrates Montana Gov. Greg Gianforte’s signing …

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Arizona Becomes the 31st State With a Rare Disease Advisory Council

Published May 13, 2025 by NORD

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New State Council Will Help Shape Health Policies for Arizonans Living with Rare Diseases PHOENIX, May 13, 2025 — The National Organization for Rare Disorders (NORD®) celebrates a significant milestone …

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Honoring Nurses on International Nurses Day: The Bridge Between Rare Disease and Hope

Published May 12, 2025 by NORD

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Patient Stories

By Alicia Truelove, MSN, RN, CV-BC, NPD-BC International Nurses Day, May 12, is a time to honor and celebrate the heart of healthcare: nurses. As a nurse of over 14 …

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#UndiagnosedLife: An Ode to My Wingman on My Diagnostic Odyssey

Published May 10, 2025 by NORD

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Patient Stories

By Christine McGarvey, NORD Rare Action Network Ambassador for Pennsylvania I have been on a long odyssey for over a decade in search of an ever-elusive diagnosis. In addition, I …

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Rare Faces of Medicaid: What’s at Stake for the Rare Community?

Published May 1, 2025 by NORD

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Congress is currently considering potentially sweeping funding cuts to Medicaid, a program that provides life-saving health care coverage and services to those who need it most. What is at stake …

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Rare Disease News

Featured News

NORD CEO Statement on the Termination of the Advisory Committee on Heritable Disorders in Newborns and Children

News & Articles

First Rare Disease Therapy Developed with a NORD IAMRARE Patient Registry Secures FDA Approval

Find Your Herd Through Running for Rare This Global Running Day

In Memory of Kate O’Bara: Raising Awareness for Vascular EDS

FDA CBER Director Dr. Vinay Prasad to Keynote NORD’s Inaugural Rare Disease Scientific Symposium, Convening Top Researchers, Industry Leaders, and Federal Regulators

In Honor of Eddie — A Story of Courage, Love, and an Unbreakable Bond

Montana Establishes Rare Disease Advisory Council to Support Residents Living with Rare Conditions

Arizona Becomes the 31st State With a Rare Disease Advisory Council

Honoring Nurses on International Nurses Day: The Bridge Between Rare Disease and Hope

#UndiagnosedLife: An Ode to My Wingman on My Diagnostic Odyssey

Rare Faces of Medicaid: What’s at Stake for the Rare Community?

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