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

Published December 21, 2024 by NORD

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For the last several years, the National Organization for Rare Disorders (NORD) has had the privilege of working in close collaboration with Members of Congress from both sides of the … Continue reading "The Needs of Rare Disease Patients Cannot Be Forgotten in this Week’s Congressional Hearing on Lab Developed Diagnostic Tests (LDTs)"...

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The Needs of Rare Disease Patients Cannot Be Forgotten in this Week’s Congressional Hearing on Lab Developed Diagnostic Tests (LDTs)

Published March 20, 2024 by NORD

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Advocacy

By Karin Hoelzer, Director of Policy and Regulatory Affairs at the National Organization for Rare Disorders (NORD) How should lab developed diagnostic tests (LDTs) be regulated? As the House Energy …

Continue reading “The Needs of Rare Disease Patients Cannot Be Forgotten in this Week’s Congressional Hearing on Lab Developed Diagnostic Tests (LDTs)”

National Organization for Rare Disorders (NORD) Releases Annual State Report Card, Revealing Trends in Rare Disease Health Care Coverage

Published March 6, 2024 by NORD

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NORD’s State Report Card grades states on policies that benefit or jeopardize health care coverage, access, and affordability for people living with rare diseases The National Organization for Rare Disorders …

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Fighting Isolation When You Are One in a Million: Gabby’s Story in Honor of Rare Disease Day

Published February 29, 2024 by NORD

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By Gabrielle Z. A very special person once told me: “You are dealt the cards in this life that only you are meant to handle.” My name is Gabrielle but …

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Living a Full Life After Diagnosis: Risa’s Story in Honor of Rare Disease Day

Published February 29, 2024 by NORD

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By Risa A. My name is Risa and I have Acromegaly. From the NORD Rare Disease Report: Acromegaly is a rare, slowly progressive, acquired disorder that affects adults, most …

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The Needs of Rare Disease Patients and the Value of Orphan Drugs Will Take Center Stage at this Week’s Rare Disease Congressional Hearing

Published February 28, 2024 by NORD

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By Heidi Ross and Karin Hoelzer What needs to be done to better support rare disease patients, and what is the value of the prescription drugs that treat rare diseases? …

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We Exist: Tanita’s Experience as a Black Woman with Huntington’s Disease

Published February 27, 2024 by NORD

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Patient Stories

By Tanita A. My name is Tanita and I am an African-American woman living with Huntington’s Disease. I did not have a family history of Huntington’s Disease; I am the …

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Challenges Are Just Waiting for Solutions: Allison’s Story in Honor of Rare Disease Day

Published February 26, 2024 by NORD

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By Allison R. My ultra-rare disease, Cryopyrin Associated Periodic Syndrome (CAPS) has been isolating, defeating, and has made me feel helpless—but not hopeless. It has caused changes of career paths …

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Overcoming the Obstacles: Sareena’s Story in Honor of Rare Disease Day

Published February 23, 2024 by NORD

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By Sareena Z. My name is Sareena and I was diagnosed with severe Factor VII deficiency when I was two months old. I am now 28 years old. Factor VII …

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New Trajectories and Everyday Struggles: Thomas’s Story in Honor of Rare Disease Day

Published February 16, 2024 by NORD

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By Thomas B. I’m a patient living with Myasthenia Gravis, a rare neuromuscular autoimmune disease that affects the voluntary muscles. The immune system kills the chemical receptors in our muscles …

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Invisible Stripes: Leigh’s Story in Honor of Rare Disease Day

Published February 9, 2024 by NORD

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By Leigh P. I was diagnosed with Graves’ disease in 2019 after four consecutive miscarriages and a lifetime of symptoms that were misunderstood by professionals. A person with Graves’ …

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