Rare Disease Day at NIH

Rare Disease Day at NIH aims to raise awareness about rare diseases, the people they affect, and collaborations that address scientific challenges and advance research for new treatments. Each year the event brings together a broad audience of patients, patient advocates, caregivers, health care providers, researchers, trainees, students, industry representatives, and government staff to share the latest information in rare diseases research.

Supported in part by NHBLI, this year’s virtual event features a special presentation by Margaret Bevans, Ph.D., R.N., director of the Office of Research Nursing in the NHLBI Office of the Clinical Director. She will discuss caregiver resilience through mind, body, and spirit. In addition, panel discussions will be held that center around the following topics:

• Diversity in Rare Diseases Research and Equity of Care
• Individualized Therapeutic Approaches and Personalized Medicine
• Successful Clinical Trial Enrollment for Pulmonary Sarcoidosis during a Pandemic
• Collaborative Research Partnerships
• Natural History Data Collection for a Gene Therapy Clinical Trial
• The Use of Telehealth During COVID-19
• Diagnostic Challenges for Rare and Undiagnosed Conditions

The event is free and open to the public. View and download the agenda.

Videocast

Watch the event via NIH Videocast.

Contact

For more information or to request special accommodations, contact Cara Lynch.

Organizing Committee

• National Center for Advancing Translational Sciences
• NIH Clinical Center
• National Cancer Institute
• National Heart, Lung, and Blood Institute
• National Institute on Alcohol Abuse and Alcoholism
• National Institute of Neurological Disorders and Stroke
• Rare Diseases Clinical Research Network’s Coalition of Patient Advocacy Groups
• U.S. Food and Drug Administration (FDA)
• The Children’s Inn at NIH
• EveryLife Foundation for Rare Diseases
• National Organization for Rare Disorders
• United BioSource LLC