Physician-reviewed information on orphan and rare diseases. Each condition affects fewer than 200,000 people in the United States. Find comprehensive resources for diagnosis, management, and patient support.
Rare (orphan) diseases are conditions affecting small populations. Diagnosis is often delayed; patient networks and registries (like Orphanet) provide crucial support. Always consult specialists and patient organizations for the latest guidance.
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International database of rare diseases and orphan drugs
Information and research resources from the US National Institutes of Health
Patient advocacy, support groups, and disease-specific information
Global community connecting patients, families, and researchers