The Genetic and Rare Diseases Information Center (GARD)
from NIH’s National Center for Advancing Translational Sciences has easy-to-understand information about rare or genetic diseases. You may also call 1-888-205-2311 (toll-free) to talk to a GARD information specialist.
The NCATS Toolkit for Patient-Focused Therapy Development
has a collection of online resources that can help patient groups advance medical research for rare diseases.
The Rare Diseases Registry Program
from NIH’s National Center for Advancing Translational Sciences offers online resources for patient groups on setting up and managing a successful registry.
The Undiagnosed Diseases Network (UDN)
is a research study funded by NIH that aims to improve the diagnosis and care of patients with undiagnosed diseases. You can contact the UDN Coordinating Center at UDN@hms.harvard.edu
or 1-844-746-4836 (toll-free).
has information about clinical studies at NIH and other institutions that are currently enrolling patients. For more information on studies conducted at NIH, contact the Patient Recruitment and Public Liaison Office at firstname.lastname@example.org
or 1-800-411-1222 (toll-free).
Rare Diseases Community Resources
: NCATS offers free materials and resources to help patients, caregivers, patient support organizations, healthcare providers, and scientists learn about rare diseases and help advance research.
The Rare Diseases Registry (RaDaR) Program
defines best practices for developing patient registries.
Learn about FDA Patient Engagement
: Understand the different opportunities in which patients and caregivers can get involved.