Don’t miss the in-person and virtual celebration of Rare Disease Day at NIH on Thursday, February 29, 2024, 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 collaborations that address scientific challenges and advance research for 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, in-person exhibitors and scientific posters, and an art exhibition.
The RDCRN will host a booth during the event. You can visit our booth at any time to learn more about the RDCRN.
In addition, several RDCRN participants will speak during the event:
- 11:05 am EST—Session 1: Artificial Intelligence (AI) and Its Potential Role in Rare Diseases
- Panelist
Manisha Balwani, MD, MS, FACMG, Principal Investigator, Porphyrias Consortium (PC); Professor and Chief, Division of Medical Genetics and Genomics, Department of Genetics and Genomic Sciences, Icahn School of Medicine at Mount Sinai
- Panelist
- 3:35 pm EST—Session 2: Recent Approvals and Advancements in Gene Therapy
- Gene Therapy for Patients with Cerebral Adrenoleukodystrophy
Florian S. Eichler, MD, Principal Investigator, Global Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN); Director, Center for Rare Neurological Diseases, Massachusetts General Hospital; Professor, Neurology, Harvard Medical School
- Gene Therapy for Patients with Cerebral Adrenoleukodystrophy
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 here. 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).
