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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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Diseases


About

GARD maintains a list of rare diseases and related terms to help people find reliable information. In the United States, a rare disease is defined as one that affects fewer than 200,000 people. The prevalence of a rare disease usually is an estimate and may change over time. Inclusion in GARD's list does not serve as official recognition by the NIH as a rare disease and should not be used to assume that a disease is rare.

GARD Diseases Landing

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

Browse by Category

GARD Diseases Landing

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

What's New?

GARD Diseases Landing

What’s New?

GARD Information Specialists have posted new information for these diseases:
  • Arrhythmogenic right ventricular cardiomyopathy
  • Oculopharyngeal muscular dystrophy
  • Autosomal recessive primary microcephaly
  • Morgagni-Stewart-Morel syndrome
  • Factor V deficiency
  • Diamond-Blackfan anemia
  • Isovaleric acidemia
  • TARP syndrome

List of FDA Orphan Drugs

GARD Diseases Landing

List of FDA Orphan Drugs

See a list of orphan products for rare diseases. New treatments are available for:
  • Erythropoietic protoporphyria
  • Tyrosinemia type 1
  • West syndrome
  • Thrombotic thrombocytopenic purpura, acquired
  • Primary liver cancer
  • Blastic plasmacytoid dendritic cell
  • Paroxysmal nocturnal hemoglobinuria
  • Merkel cell carcinoma

Ask us a Question

If you can’t find your condition or need more information, contact an Information Specialist at the Genetic and Rare Diseases Information Center (GARD). We cannot make a diagnosis or give medical advice, but we can provide information.
Call our toll-free number

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See more ways to Contact GARD

GARD Information Navigator

Try our interactive tool for help finding information, services, experts, financial aid, and more.

GARD Information Navigator
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