Rare Disease News

Breaking Down Data Siloes: New Analytics Platform Sparks Opportunity for Rare Disease Field

Published September 1, 2021 by NORD

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TUCSON, Ariz. and WASHINGTON, September 1, 2021 — For two years, Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD) and the U.S. Food and Drug Administration (FDA) have joined …

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Breakthrough Summit 2021: The Most Important Rare Disease Conference of the Year

Published August 27, 2021 by NORD

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August 27, 2021 – Registration is now open for the NORD Rare Diseases and Orphan Products Breakthrough Summit®. The NORD Summit, one of the largest global events in rare disease, brings together experts …

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Cure MLD, The Calliope Joy Foundation and NORD Launch A Research and Engagement Study of Metachromatic Leukodystrophy (MLD)

Published August 16, 2021 by NORD

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Research study is open to participants worldwide to advance understanding and treatments for Metachromatic Leukodystrophy, a recessive genetic disorder that is characterized by the accumulation of fats (lipids) called sulfatides …

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NORD Named an Official Charity Partner of the 2021 TCS New York City Marathon

Published July 15, 2021 by NORD

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Washington, DC – The National Organization for Rare Disorders® (NORD) was named an Official Charity Partner for the 2021 TCS New York City Marathon, taking place on November 7. This year serves …

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NORD Issues Statement on California v. Texas Ruling

Published June 17, 2021 by NORD

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Washington, DC, June 17, 2021—The following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD) regarding today’s US Supreme Court decision …

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NORD Welcomes Edward Neilan, MD, PhD, as Chief Medical and Scientific Officer

Published May 27, 2021 by NORD

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Quincy, MA and Danbury, CT, May 27, 2021—The National Organization for Rare Disorders (NORD®), the leading patient advocacy organization dedicated to the identification, treatment and cure of rare diseases, announced …

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NORD Announces New Board Chair and New Member of its Board of Directors

Published May 18, 2021 by NORD

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Washington, DC, May 18, 2021—The National Organization for Rare Disorders (NORD®) announced that during a recent Board of Directors meeting, Kay Holcombe was elected as Chair and Dennis Jackman joined …

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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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CDISC and NORD Partner to Develop Data Standards for Rare Diseases

Published April 22, 2021 by NORD

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Danbury, CT and Austin, TX – April 22, 2021 – CDISC and the National Organization for Rare Disorders (NORD®) have announced a partnership to develop global data standards for rare …

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New Study Investigates the Number of Available Orphan Products, Generics and Biosimilars

Published March 25, 2021 by NORD

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Washington, DC, March 25, 2021—The National Organization for Rare Disorders (NORD®) today announced the findings of a new study that details the number of orphan products, generics and biosimilars available to treat rare diseases. NORD commissioned Avalere to conduct the analysis to examine if laws and …

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