Rare Disease News

News & Articles

Quintissa’s Rare Disease Story in Honor of World Blood Donor Day

Published June 14, 2023 by NORD

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Patient Stories

By Quintissa P. My name is Quintissa and I was born with sickle cell anemia. As a result of this condition, my hematologist has estimated that I’ve received well over …

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National Organization for Rare Disorders Urges Congress To Protect Funding for Rare Disease Research, Commends Exclusion of Work Requirements for Medicaid in Debt Ceiling Deal

Published June 2, 2023 by NORD

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Washington, DC, June 2, 2023 – This week, the Senate passed a debt ceiling deal (HR 3746) by a 63-36 vote and which now goes to President Biden for his …

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The Healing Power of Movement: Lena’s Rare Disease Story

Published May 26, 2023 by NORD

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Patient Stories

By Lena Z. I was born with two rare diseases: medullary sponge kidneys (MSK), a birth defect where changes occur in the tiny tubes inside a fetus’ kidneys, and hypermobile Ehlers-Danlos …

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Recognizing Abbey Meyers, NORD Founder and Mother of a Movement

Published May 12, 2023 by NORD

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On this Mother’s Day, NORD would like to recognize the rare disease “Mom” who 40 years ago, brought us all together to where we are today. Abbey Meyers is the …

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National Network of NORD Rare Disease Centers of Excellence Expands to 40 U.S. Medical Institutions

Published May 4, 2023 by NORD

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NORD announced nine new research hospitals have been designated NORD Rare Disease Centers of Excellence, joining a network of thousands of experts committed to sharing knowledge to solve the greatest …

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Maryland Governor Signs Legislation to Address Needs of Rare Disease Patients and Families Throughout the State

Published May 3, 2023 by NORD

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More than Half of U.S. States Now Solicit Input of State Rare Disease Advisory Councils to Inform Local Health Policies May 3, 2023, Annapolis, MD – Today, patients and families in …

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Undiagnosed: When Your Child Is Misdiagnosed

Published April 29, 2023 by NORD

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Patient Stories

By Jessica F. This story is about Sophie, my now 10-year-old daughter who is impacted by limb girdle muscular dystrophy type 2A/R1, previously misdiagnosed and treated for necrotizing myositis. Sophie’s …

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Undiagnosed: When Your “Depression” Isn’t Depression

Published April 29, 2023 by NORD

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Patient Stories

By Kylee S. This story is about me and my long journey to a diagnosis. Although I might have had mild symptoms as a child, it was nothing noticeable. My …

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Undiagnosed: When Your Rare Diagnosis Comes 30 Years Late

Published April 29, 2023 by NORD

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Patient Stories

By Rachel O. I was born rare, but my zebra journey did not begin until just five years ago at the age of 32. Throughout those 32 years, I faced signs …

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Congress should protect the intent of the Orphan Drug Act and pass the RARE Act

Published April 24, 2023 by NORD

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Advocacy

Rare disease patients, including children, deserve safe and effective therapies. By Karin Hoelzer, Director of Policy and Regulatory Affairs at NORD This year marks the 40th anniversary of the Orphan …

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Rare Disease News

Featured News

Statement by National Organization for Rare Disorders CEO, Pamela Gavin, on Congress’s Exclusion of Critical Rare Disease Bills in the Continuing Resolution

News & Articles

Quintissa’s Rare Disease Story in Honor of World Blood Donor Day

National Organization for Rare Disorders Urges Congress To Protect Funding for Rare Disease Research, Commends Exclusion of Work Requirements for Medicaid in Debt Ceiling Deal

The Healing Power of Movement: Lena’s Rare Disease Story

Recognizing Abbey Meyers, NORD Founder and Mother of a Movement

National Network of NORD Rare Disease Centers of Excellence Expands to 40 U.S. Medical Institutions

Maryland Governor Signs Legislation to Address Needs of Rare Disease Patients and Families Throughout the State

Undiagnosed: When Your Child Is Misdiagnosed

Undiagnosed: When Your “Depression” Isn’t Depression

Undiagnosed: When Your Rare Diagnosis Comes 30 Years Late

Congress should protect the intent of the Orphan Drug Act and pass the RARE Act

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