Don’t miss the in-person and virtual celebration of Rare Disease Day at NIH on Tuesday, February 28, 2023, from 9 am to 5 pm EST. Each year, the NIH’s National Center for Advancing Translational Sciences (NCATS) and the NIH Clinical Center sponsor the event as part of the global Rare Disease Day observance. Goals include raising awareness about rare diseases, the people they affect, and NIH research collaborations underway to address scientific challenges and advance new treatments.
The event will be held in person at NIH Main Campus (Natcher Conference Center). There also will be a virtual livestream via NIH VideoCast with the event archived for replay afterward. The event agenda features panel discussions, rare diseases stories, exhibitors, and scientific posters.
RDCRN will host a virtual booth space and meeting session during the event. You can visit our booth at any time to learn more about the RDCRN. During networking breaks at 11:20 am, 12:50 pm, and 2:50 pm EST, RDCRN staff will be available to chat and answer questions.
In addition, several RDCRN participants will speak during the event:
- 12:35 pm EST: Rare Story #2: Care Transition Challenges for Young Adults with Rare Diseases — A Patient and Provider Perspective
- Cary O. Harding, MD, Principal Investigator, Phenylalanine Families and Researchers Exploring Evidence (PHEFREE) Consortium, Rare Diseases Clinical Research Network (RDCRN); Professor, Molecular and Medical Genetics, School of Medicine, Oregon Health & Science University
- Brittany M. Holmes, MSN, APRN, FNP-BC, Rare Disease Patient; Advisor, PHEFREE Consortium, RDCRN; Metabolism Nurse Practitioner, Division of Genetics and Genomics, Boston Children’s Hospital
- 3:40 pm EST: Rare Story #6: Scientific and Patient Journey to the First US Food and Drug Administration–Approved Drugs for Two Rare Eosinophilic Diseases
- Marc E. Rothenberg, MD, PhD, Principal Investigator, Consortium of Eosinophilic Gastrointestinal Disease Researchers (CEGIR), RDCRN; Director, Division of Allergy and Immunology, Cincinnati Children’s Hospital Medical Center (CCHMC); Director, Cincinnati Center for Eosinophilic Disorders, CCHMC; Professor, University of Cincinnati Department of Pediatrics
- Ellyn Kodroff, Coalition of Patient Advocacy Groups (CPAG) Representative, CEGIR, RDCRN; President and Founder, Campaign Urging Research for Eosinophilic Disease (CURED)
- 4 pm EST: Session 4: Rare Diseases Therapeutics and the Role of Advocacy and Industry Collaborations
- Kristen Wheeden, MBA, Rare Disease Caregiver; CPAG Representative, Porphyrias Consortium, RDCRN; President, United Porphyrias Association
Rare Disease Day at NIH is free and open to the public, including patients, patient advocates, health care providers, researchers, trainees, students, industry representatives, and government employees. Register in advance at this link. We hope to see you there!
The Rare Diseases Clinical Research Network (RDCRN) is funded by the National Institutes of Health (NIH) and led by the National Center for Advancing Translational Sciences (NCATS) through its Division of Rare Diseases Research Innovation (DRDRI). Now in its fourth five-year funding cycle, RDCRN is a partnership with funding and programmatic support provided by Institutes, Centers, and Offices across NIH, including the National Institute of Neurological Disorders and Stroke, the National Institute of Allergy and Infectious Diseases, the National Institute of Diabetes and Digestive and Kidney Diseases, the Eunice Kennedy Shriver National Institute of Child Health and Human Development, the National Institute of Arthritis and Musculoskeletal and Skin Diseases, the National Heart, Lung, and Blood Institute, the National Institute of Dental and Craniofacial Research, the National Institute of Mental Health, and the Office of Dietary Supplements.
