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Tags: Cincinnati Children's Hospital Medical Center, Hemophilia Federation of America, Networking, NORD Rare Summit, NORD Summit, Poster Abstracts, Rare Diseases and Orphan Products Breakthrough Summit, Sanofi Genzyme
September 11, 2018
TOPIC:
Featured News, Press Releases, Research
Posted by Laura Mullen
Washington, D.C., September 11, 2018—The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, has announced the addition of ten new rare disease registries, made possible through a multi-year grant award from Shire.
NORD’s IAMRARETM Registry Program provides a web-based tool to collect patient experience and patient-reported outcomes data across… Read More
Tags: clinical trials, diagnostics, FDA, IAMRARE, IAMRARE Registry Program, longitudinal, National Organization for Rare Disorders, natural history study, NORD, Pamela Gavin, patient-centric, registries, Shire, standards of care, treatment protocols
September 6, 2018
TOPIC:
Featured News
Posted by Jennifer Huron
2018 is a big year for the rare disease community – NORD is celebrating its 35th anniversary. For the past 35 years, NORD has taken pride in being the rare disease community’s biggest advocate. Our policy, research and other efforts have played a significant role over the years, advocating for what matters most… Read More
Tags: #DoYourShare, 21st Century Cures, 35th Anniversary, Advancing Hope Act, Affordable Care Act, American Medical Student Association, Citizen's Petition, CME, continuing medical education, drug pricing, Eugene Washington Engagement Award, FDA, FDA Safety and Innovation Act, FDA user fees, Finding a Zebra Among Many Horses, Food and Drug Administration Reauthorization Act of 2017, Gene therapy, Good Morning Peyton, IAMRARE, Janet Woodcock, Medicaid, medical devices, Medical Foods, medical foods coverage, natural history studies, newborn screening, NIH, NORD Physician Guides, NORD Research Grant Program, NORD Summit, Orphan Drug Act, orphan drug tax credit, patient registries, Patient-Centered Outcomes Research Institute, Patient/Caregiver Speakers Bureau, PCORI, prescription cost sharing requirements, President Trump, QuintilesIMS Institute, Rare Action Network, Rare and Neglected Diseases Caucus, Rare Disease Congressional Caucus, Rare Pediatric Disease Priority Review Voucher, Rare Pediatric Disease Priority Review Voucher Program, RareInsights, registries, State Report Card, Student Membership, TRICARE, University of Maryland, user fees, zebra, Zika
September 5, 2018
TOPIC:
Events, Featured News
Posted by Laura Mullen
One of the unique aspects of the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit is its theme, “A New Era of Patient-Focused Innovation,” which we will be examining from a variety of perspectives. Kicking off the Summit will be the Patient/Caregiver Opening Address featuring a panel of young rare disease patient advocates sharing their hopes, thoughts… Read More
Tags: adrenoleukodystrophy, ALD, American University of Integrative Sciences School of Medicine, bicycle, Christopher Anselmo, Dr. Anita Gupta, Gabriel Low, Harjot Randhawa, hypokalemic periodic paralysis, Miyoshi myopathy/limb-girdle muscular dystrophy type 2, Remember the Girls, Taylor Kane
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