Rare Disease News

Protecting Patients and Telehealth Access: Over 230 National Organizations Urge Governors Across the Country to Maintain and Expand Licensure Flexibilities Throughout Federal Public Health Emergency

Published November 1, 2021 by NORD

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Letter to Governors Stresses Urgency of Expiring Licensure Waivers and its Impact on Patient Access to Care Washington, DC – November 1, 2021 – Today, more than 230 organizations sent …

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NORD Response to New Draft of the Build Back Better Act

Published October 29, 2021 by NORD

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The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, issued the following statement in response to newly introduced draft …

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NIH, FDA and 15 private organizations join forces to increase effective gene therapies for rare diseases

Published October 27, 2021 by NORD

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October 27, 2021 – The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for …

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Record-Setting NORD Breakthrough Summit Welcomes Nearly 1,000 Members of the Rare Disease Community

Published October 21, 2021 by NORD

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Two days of insightful and engaging speakers, sessions, and networking at the most important event in rare disease saw record-breaking attendance October 21, 2021 – This week, the National Organization for Rare Disorders (NORD®) hosted the virtual Rare Diseases and Orphan Products …

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Rare Cancer Day 2021 Brings Together Rare Disease Community and Advocates

Published September 29, 2021 by NORD

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Washington, DC, September 29, 2021— Tomorrow, the rare disease community will celebrate the third annual Rare Cancer Day. Spearheaded by the National Organization for Rare Disorders (NORD®) Rare Cancer Coalition™, Rare Cancer Day is …

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NORD Welcomes Aliza Fink, D.Sc. as Director of Research Programs

Published September 23, 2021 by NORD

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Washington, DC, September 23, 2021—The National Organization for Rare Disorders (NORD®), the leading patient advocacy organization dedicated to the identification, treatment and cure of rare diseases, announced the appointment of …

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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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Rare Disease Innovation and Data Sharing Accelerated by New RDCA-DAP Program

Published September 15, 2021 by NORD

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TUCSON, Ariz. and WASHINGTON, September 15, 2021 — The Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP®) officially launched its next phase on Tuesday establishing itself as the leading platform to …

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NORD Statement on Proposed Changes to the Orphan Drug Tax Credit

Published September 13, 2021 by NORD

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The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the more than 25 million Americans with rare diseases, issued the following statement in response to upcoming …

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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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