Rare Disease News

Musical Act Announced for Rare Impact Awards on May 18th

Published March 2, 2017 by NORD

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Nationally touring musical artist Gaelynn Lea will perform at NORD’s Annual Rare Impact Awards.

NORD Issues Statement on President Trump’s Address to Congress

Published March 1, 2017 by NORD

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NORD Issues Statement on President Trump’s Address to Congress Washington, D.C., March 1, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans …

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NORD Unveils “Do Your Share for Rare,” Year-long Campaign for Rare Disease Public Awareness

Published February 28, 2017 by NORD

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Danbury, Conn., February 28, 2017—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, announced a year-long awareness campaign for …

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NORD Awards New Research Grants for Rare Disease Research

Published February 24, 2017 by NORD

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Research Grants are available from NORD for the study of rare diseases. The deadline to send in initial applications is Friday, June 23.

NORD Publishes Report on Spontaneous Intracranial Hypotension

Published January 27, 2017 by NORD

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New Resource Aims to Provide Information for Patients, Families and the Public Washington, D.C., January 27, 2017—As part of its ongoing series to promote awareness and education of rare diseases, …

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Orphan Drugs Represent 41 Percent of All New Medications

Published January 11, 2017 by NORD

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Washington, D.C., January 11, 2017—Data released by the U.S. Food and Drug Administration (FDA) show that 9 of the 22 new medications approved in 2016, or 41 percent, are orphan drugs to …

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First Natural History Registry for Necrotizing Enterocolitis

Published January 9, 2017 by NORD

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Naugatuck, CT, and Danbury, CT, January 09, 2017—The Morgan Leary Vaughan Fund (Morgan’s Fund) has launched the first natural history registry for Necrotizing Enterocolitis (NEC)–an inflammatory disease that leads to …

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Largest-Ever Study of SYNGAP1 (MRD5), Linked to Autism, was Launched

Published December 23, 2016 by NORD

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Research study is open to participants worldwide to advance understanding and treatments for rare disease causing non-syndromic intellectual disability ranging from mild to severe with attention deficits, impulsivity, and/or mood …

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President Signs 21st Century Cures Medical Innovation Bill Into Law

Published December 13, 2016 by NORD

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Today, President Obama signed into law the 21st Century Cures Act, a game-changing bill for medical innovation. The bill includes many provisions that will improve the discovery, development, and delivery …

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State Report Card to Help People with Rare Diseases

Published December 13, 2016 by NORD

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Washington, D.C., December 13, 2016—A majority of states are not measuring up on legislative solutions that reduce the burden of rare diseases affecting 30 million Americans, according to a new …

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