Database for studies, reviews, and related genes/diseases.
Search for authors or researchers working on your gene.
Provides online resources to help patient groups with research, registries, and clinical trial readiness.
Advances research, advocacy, education, and patient assistance. Provides resources for diagnosis, helps patients find specialists, and offers financial assistance for medications and travel.
Advances medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment, and data sharing.
A network of pediatric health systems that connects to disease registries, longitudinal observational studies, and clinicians and scientists.
Track ongoing clinical trials and investigational therapies.
A multi-day forum to provide parents and other patient advocates seeking to develop rare disease therapies with an opportunity to connect with their peers and to learn from rare disease drug development experts.
Allows foundations to see if there are government grants related to their condition and may help connect them to researchers.
Brings together scientists, physicians, patient advocates, and other stakeholders focused on genetic and cellular therapies.
Non-profit providing policy and science support, guidance, and connections to advocacy programs.
Provides free access to information, diagnosis, and resources.
Search to discover if cell lines already exist for a specific disease.
Provides high-quality information on rare diseases and ensures equal access to knowledge for all stakeholders. Maintains the Orphanet rare disease nomenclature (ORPHAcode).
A leader in driving U.S. clinical research to be more patient-centered, which results in evidence that is more relevant and useful.
Expert, personalized guidance and operational support to ultra-rare disease foundations at every stage of development.
Provides rare disease advocates with the tools, training, and trust to lead – to activate communities, advance research, and widen the drug development pipeline.
Disease registries/advocacy networks
Find more specific knowledge, build patient cohorts, and connect to researchers. Look for disease nonprofits, NIH/ORDR (Office of Rare Diseases) programs, or academic consortia.
Consulting networks
Networks like RARE Science and CureGRIN connect patient-led groups to experienced biotech mentors.
Disease-specific foundations
These organizations often share or mentor through advisory boards (e.g., Global Genes RARE-X, EveryLife Foundation).
A leader in driving U.S. clinical research to be more patient-centered, which results in evidence that is more relevant and useful.
