On September 8, the U.S. Department of Health and Human Services announced the selection of four new senior leaders at the U.S. Food and Drug Administration (FDA), three of which…
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August 13, 2026 — A National Newborn Screening Workgroup was announced by the Health Resources and Services Administration (HRSA) via public website (pending Federal Register publication on August 12, 2026).…
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NORD Submits Formal Comments to CMS Urging Changes to Align the Final Rule with Congressional Intent and Protect Medically Frail People with Rare Diseases NORWELL, Mass., July 31, 2026 —…
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Clayton, MO, April 19, 2022 —Today, April 19, 2022, The Snow Foundation for Wolfram Syndrome Research and the National Organization for Rare Disorders, Inc. launched the largest-ever study to research …
Read moreDr. Adrienne Hammill is Research Director of the Hemangioma & Vascular Malformation Program and Director of the HHT Center of Excellence at Cincinnati Children’s Hospital. Her path to becoming a …
Continue reading “One Piece Of The Puzzle: Dr. Adrienne Hammill”
Read more90 Patient Organizations Sign-on to NORD Letters to Secretary Becerra and Congressional Leaders on Strengthening the Accelerated Approval Pathway April 8, 2022, Washington, DC – This week, the National Organization …
Read moreKari Luther Rosbeck is the President & Chief Executive Officer of the TSC Alliance, a nonprofit whose mission is to find a cure for Tuberous Sclerosis Complex (TSC) while improving …
Continue reading “From Pain To Purpose: Kari Luther Rosbeck”
Read moreChris Anselmo is the Director of Market Intelligence at the Muscular Dystrophy Association but he never envisioned becoming an advocate for a disease he never thought he’d get. After a …
Continue reading “Chris Anselmo: The Accidental Muscular Dystrophy Advocate”
Read moreNORD’s Rare Impact Award ceremony on June 26 will honor all those making strides to improve the lives of patients and families in the rare disease community Cleveland, OH, March …
Read moreFrom the time I was three years old, my life has centered around horses. When I was diagnosed with my rare disease, diffuse scleroderma, my first thought was “will I …
Continue reading “Scleroderma and Horses: Whitney’s Rare Disease Day Story”
Read moreI am a 16-year-old pediatric patient who has been struggling with Sjögren’s for years. There are so many ways my condition has affected my life in both bad and surprisingly …
Continue reading “Rare Disease Day 2022: Grace’s Story”
Read moreI am a mom, advocate, fighter and the biggest support to my two-year-old son Braxton, who was born with Phenylketonuria (PKU). My biggest mission since my son was born was …
Continue reading “#ShowingYourStripes Through Advocacy: Kala’s Story for Rare Disease Day”
Read moreI was born with a rare genetic condition known as Congenital Insensitivity to Pain (CIP), which leaves an individual unable to sense physical pain. This “disease” or condition, as I …
Continue reading “Removing the Obstacles: Steven’s Rare Disease Day Story”
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