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U.S. Department of Health & Human Services
National Institutes of Health
NCATS
National Center for Advancing and Translational Sciences National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences
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  • Browse A-Z
  • Find Diseases By Category expand submenu for Find Diseases By Category
    • Autoimmune / Autoinflammatory diseases
    • Bacterial infections
    • Behavioral and mental disorders
    • Blood Diseases
    • Chromosome Disorders
    • Congenital and Genetic Diseases
    • Connective tissue diseases
    • Digestive Diseases
    • Ear, Nose, and Throat Diseases
    • Endocrine Diseases
    • Environmental Diseases
    • Eye diseases
    • Female Reproductive Diseases
    • Fungal infections
    • Heart Diseases
    • Hereditary Cancer Syndromes
    • Immune System Diseases
    • Kidney and Urinary Diseases
    • Lung Diseases
    • Male Reproductive Diseases
    • Metabolic disorders
    • Mouth Diseases
    • Musculoskeletal Diseases
    • Myelodysplastic Syndromes
    • Nervous System Diseases
    • Newborn Screening
    • Nutritional diseases
    • Parasitic diseases
    • Rare Cancers
    • RDCRN
    • Skin Diseases
    • Viral infections
  • List of FDA Orphan Drugs
  • GARD Information Navigator
  • FAQs About Rare Diseases


GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet.

pills fda orphan drugs
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No hemos publicado información en español sobre enfermedades que comiencen con esta letra. Por favor contáctenos.

F
  • Fabry disease
  • Factor VII deficiency
  • Factor X deficiency
  • Factor XIII deficiency
  • Fallopian tube cancer
  • Familial cold autoinflammatory syndrome
  • Familial colorectal cancer
  • Familial hypercholesterolemia
  • Familial hypertrophic cardiomyopathy
  • Familial isolated hyperparathyroidism
  • Familial Mediterranean fever
  • Familial prostate cancer
  • Familial pulmonary arterial hypertension leucopenia and atrial septal defect
  • Familial transthyretin amyloidosis
  • Familial ventricular tachycardia
  • Fibrinogen deficiency, congenital
  • Focal dystonia
  • Follicular lymphoma
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