Rare Disease News

NORD Launches First Canadian Patient Registries on Its IAMRARE Platform

Published May 10, 2024 by NORD

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The National Organization for Rare Disorders (NORD) announced the launch of two Canadian patient registries within its IAMRARE® Program, in partnership with the Children’s Hospital of Eastern Ontario (CHEO) Research …

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All Things Kabuki and NORD® Launch Natural History Study of Kabuki Syndrome

Published August 3, 2022 by NORD

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Wasilla, Alaska, August 3, 2022—All Things Kabuki and the National Organization for Rare Disorders, Inc. have launched a study with global reach to research Kabuki syndrome, which causes a variety …

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The Snow Foundation for Wolfram Syndrome Research and NORD® Launch Natural History Study of Wolfram Syndrome

Published April 19, 2022 by NORD

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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 …

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Tatton Brown Rahman Syndrome Community and NORD® Launch Natural History Study of Tatton Brown Rahman Syndrome (TBRS)

Published September 22, 2021 by NORD

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Research study is open to participants worldwide to advance understanding and treatments for TBRS, a mutation of DNMT3A, causing overgrowth, autism, intellectual disabilities, orthopedic concerns, cardiac issues, and a variety …

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AAMDSIF and NORD Launch New Natural History Study of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Published May 6, 2021 by NORD

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Bethesda, MD, May 6, 2021—The Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD)® today launched the largest-ever study to research Paroxysmal Nocturnal Hemoglobinuria …

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ACD and NORD Launch the CreatineInfo Registry and Natural History Study of Cerebral Creatine Deficiency Syndromes

Published March 22, 2021 by NORD

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Carlsbad, CA (March 15, 2021)— The Association for Creatine Deficiencies (ACD) and the National Organization for Rare Disorders (NORD) launched the largest-ever study to research Cerebral Creatine Deficiency Syndromes (CCDS). …

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The Cute Syndrome Foundation and NORD Launch The Cute Syndrome Foundation Global SCN8A Survey Series

Published March 22, 2021 by NORD

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The Cute Syndrome Foundation and the National Organization for Rare Disorders today launched a survey series to better understand patients with SCN8A mutations that cause epilepsy, movement disorders, behavioral challenges, …

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