Patients & Members – 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 24, 2020

TOPIC: Featured News, Medical, Patients & Members

NORD and Partners Publish Abstract Based on Web-Based Hemophilia A Educational Initiative for Patients, Caregivers and HCPs

Posted at November 11, 2020 08:35 am by Katie Kowalski

The National Organization for Rare Disorders (NORD®), in partnership with PlatformQ Health, the Hemophilia Federation of America and the National Hemophilia Foundation, is pleased to announce the publication of an outcomes-evaluation abstract in conjunction with the 62nd American Society of Hematology (ASH) Annual Meeting. Assessing Patient and Provider Perspectives, Clinical Practice, Behaviors, and Knowledge on Hemophilia A Care is… Read More

Tags: hematology, Hemophilia Federation of America, CME, continuing medical education, hemophilia A, PlatformQ Health, National Hemophilia Foundation, ASH, American Society of Hematology Annual Meeting, Blood

November 13, 2020

TOPIC: Featured News, Patients & Members, Advocacy, Head of the Herd

Head of the Herd: Charlene Son Rigby, President and Cofounder, STXBP1 Foundation

Posted at November 11, 2020 08:51 am by Laura Mullen

At the Head of the Herd is… Charlene Son Rigby, President and Cofounder, STXBP1 Foundation

Charlene advocates for those affected by… STXBP1 encephalopathy

Charlene is located in… San Francisco, CA

Charlene most frequently checks her… Twitter account @charleneson

How Charlene got here… My daughter, Juno, was diagnosed with STXBP1 encephalopathy in 2016 after a three-year journey to find an explanation for her condition…. Read More

Tags: STXBP1 Foundation, Charlene Son Rigby, STXBP1, STXBP1 Move to Cure, AAC device, dyslexia, whole exome sequencing, STXBP1 encephalopathy

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

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