Research Archives - NORD (National Organization for Rare Disorders)

December 3, 2020

TOPIC: Featured News, Patients & Members, Research, Advocacy, RDCA-DAP

New Video Series Featuring Patient Advocacy Group Leaders, “The Importance of Patient-Reported Data to Progress in Rare Disease,” Launched by NORD, C-Path

Posted at December 12, 2020 10:46 am by Laura Mullen

As the primary benefactors of drugs that treat rare diseases, patients and caregivers must play a role in the drug development process. Patients and caregivers bring a human perspective to the drug development process and can guide researchers toward beneficial treatment outcomes. Hear from Davelyn Hood (Board President, Congenital Hyperinsulinism International and PI, CHI… Read More

Tags: NORD, VHL Alliance, rare disease treatments, National Organization for Rare Disorders, patient advocacy, Congenital Hyperinsulinism International, Rare Disease Cures Accelerator Data and Analytics Platform, RDCA-DAP, C-Path, Critical Path Institute, Foundation for Prader-Wili Research

November 19, 2020

TOPIC: Press Releases, Featured News, Research

NORD Releases Report on the State of Rare Disease Diagnosis, Care and Treatment in America Over Three Decades

Posted at November 11, 2020 09:13 am by Laura Mullen

Washington, DC—November 19, 2020: Today, the National Organization for Rare Disorders (NORD®) published a comprehensive report through its RareInsights™ initiative: Barriers to Rare Disease Diagnosis, Care, and Treatment in the US: A 30-year Comparative Analysis. As part of an ongoing mission to generate evidence and data to advanceRead More

Tags: NORD, National Organization for Rare Disorders, Vanessa Boulanger, RareInsights, National Commission on Orphan Diseases, Director of Research, Barriers to Rare Disease Diagnosis, Care, and Treatment in the US: A 30-year Comparative Analysis

November 11, 2020

TOPIC: Featured News, Patients & Members, Research, RDCA-DAP

NORD & C-Path Present 3-Part Video Series on Challenges of Rare Disease Drug Development, How RDCA-DAP Can Help

Posted at November 11, 2020 09:15 am by Laura Mullen

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 many challenges throughout this process. The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is being developed by NORD and the Critical Path Institute (C-Path) to help overcome these… Read More

Tags: RDCA-DAP, C-Path, Critical Path Institute, rare disease drug development, rare disease cures, The Rare Disease Cures Accelerator-Data and Analytics Platform

November 10, 2020

TOPIC: Press Releases, Featured News, Patients & Members, Research

NORD Drives Rare Disease Progress Through Patient-Powered Data With IAMRARE™ Registry Members

Posted at November 11, 2020 09:48 am by Laura Mullen

Washington, DC, November 10, 2020—This year, in conjunction with its rare disease community partners, the National Organization for Rare Disorders (NORD®) continued to drive research and innovation through the IAMRARE™ registry program. Having launched its first study in 2014, today IAMRARE supports longitudinal data collection efforts for over 40 rare… Read More

Tags: NORD, Rare Diseases and Orphan Products Breakthrough Summit, metachromatic leukodystrophy, Vanessa Boulanger, IAMRARE, Inc., Aplastic Anemia and MDS International Foundation, Stephen Hahn, KrabbeConnect, Appendix Cancer/Pseudomyxoma Peritonei Research Foundation, A Cure in Sight, necrotizing enterocolitis, US Food and Drug Administration Commissioner, Gorlin Syndrome Alliance, The Moebius Syndrome Foundation, Tatton Brown Rahman Syndrome Community, COPA Syndrome Foundation, Helping Hands for GAND, Sara's Cure, The Snow Foundation, The Association for Creatine Deficiencies, The Cute Syndrome Foundation, undiagnosed patients, arachnoiditis, moyamoya

November 2, 2020

TOPIC: Featured News, Patients & Members, Research, Advocacy, Industry

NORD and Patient Advocacy Groups Host an EL-PFDD Meeting on Krabbe Disease

Posted at November 11, 2020 02:25 pm by Debbie Drell

One of the most important gatherings of the Krabbe disease community occurred in collaboration with the US Food and Drug Administration (FDA) on October 29, and it was 100% virtual with live, interactive components. FDA regulators, academic researchers and industry drug developers came together with Krabbe disease patients, caregiversRead More

Tags: Krabbe disease, Voice of the Patient, KrabbeConnect, Partners for Krabbe Research, galactocerebrosidase, The Legacy of Angels Foundation, Hunter's Hope, Gain Therapeutics, Magenta Therapeutics, PassageBio, Neurogene, lysosomal storage disorder

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