Rare Disease News

A Cure In Sight, in partnership with the UCSF Ocular Oncology Center and NORD, Launches Landmark Ocular Melanoma Natural History Study

Published September 28, 2020 by NORD

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Cleveland, OH, September 28, 2020—A Cure in Sight and the National Organization for Rare Disorders have launched an innovative study to research ocular melanoma, a disease that can cause blindness, …

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NORD and C-Path Release Video on Rare Disease Challenges in Each Step of the Drug Development Process

Published September 23, 2020 by NORD

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There are over 7,000 rare diseases, approximately 90% do not have FDA-approved treatments, and the process of developing therapies for rare diseases involves special challenges and considerations. NORD and the Critical Path Institute (C-Path) are working together …

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NORD and KrabbeConnect Launch KrabbeCURES to Study Globoid Cell Leukodystrophy

Published September 21, 2020 by NORD

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Krabbe Community United Research and Engagement Study (KrabbeCURES) We are pleased to announce the launch of KrabbeCURES, a collaborative effort between KrabbeConnect and the National Organization for Rare Disorders (NORD) …

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2020 NORD Summit to Feature Leading Experts in Public Policy, Patient Advocacy, Rare Disease Research and Regulatory Science

Published September 10, 2020 by NORD

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Washington, DC, September 10, 2020–At a time when it is vitally important for the rare disease community to come together, the National Organization for Rare Disorders (NORD®) has announced a program of …

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New Video Series on the 5 Steps of the Drug Development Process and How RDCA-DAP Can Help

Published August 27, 2020 by NORD

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There are five steps in the drug development process, which are designed to help ensure that potential new therapies are both safe and effective. Developing medical products for rare diseases encounters …

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NORD CEO Affirms Science Should Guide FDA’s Work

Published August 25, 2020 by NORD

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“Since 1906, FDA’s dedicated career employees have worked tirelessly to ensure that Americans have access to the safest supply of products we rely on every day, including food, drugs and …

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NORD’s Jayne Holtzer Rare Disease Research Grants Program Continues Its Commitment to Rare Disease Research with New Funding Opportunities

Published June 22, 2020 by NORD

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Washington, DC, June 22, 2020 – The National Organization for Rare Disorders® (NORD), the leading independent nonprofit organization representing over 25 million Americans living with rare diseases, today announced the …

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New video from NORD and C-Path addresses the question: “What is a Registry?”

Published June 2, 2020 by NORD

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What is a registry and why are they so important to the rare disease community? Together, NORD and the Critical Path Institute (C-Path) created this video to explain the value …

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NORD Awards Eleven New Grants in Eight Disease States for Rare Disease Research

Published May 19, 2020 by NORD

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Washington, DC, May 19, 2020—The National Organization for Rare Disorders (NORD®) has announced new awards through its Rare Disease Research Grant Program, which provides funding to qualified researchers for translational …

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NORD to Host an Externally-led Patient-Focused Drug Development Virtual Meeting on Krabbe Disease, Thursday, October 29

Published May 13, 2020 by NORD

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NORD, KrabbeConnect and Partners for Krabbe Research invite you to register for an externally-led Patient-Focused Drug Development (EL-PFDD) virtual meeting on Krabbe disease, taking place Thursday, October 29, 12:30-4:30pm ET. …

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