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General Information on Rare Diseases
Links to information and databases of rare diseases around the world.

Support Groups for Rare Diseases
Links to directories/databases of support and advocacy groups and organizations for rare diseases. Support groups are also listed with their specific diseases on this site.

Orphan Drugs
Articles and information on orphan drugs (drugs to treat rare diseases).

Autoimmune Disorders
Disorders in which the body in essence turns on itself and destroys its own cells.

Blood disorders
Descriptions of problems with the things that make up blood, such as red blood cells, white cells, platelets, plasma, and enzymes that control what the blood does. Links to sites offering information and support.

Book Lists/Reviews
Recommended books about or related to rare diseases and disorders.

Cancers, rare
Cancer can develop in almost any kind of cell in the body--muscle, bone, the organs, skin, and tissues. Links to organizations offering information and support are grouped by body system affected.

Chromosomal disorders
Links to information and support sites for syndromes that occur with defects in any of the 23 pairs of human chromosomes.

Clinical trials
Links to sites offering information on participation in clinical trials, such as what questions to ask and why, and lists of current studies looking for volunteers.

Digestive disorders
Rare diseases/disorders of the digestive system.

Eye Disorders
Rare conditions affecting the eyes.

Genetic - gen info
Links to general information and resources for genetic disorders. For specific disorders, search for the topic on the site.

Infectious Diseases
Diseases caused by bacteria and viruses.

Kidney/Urinary Disorders
Disorders affecting the kidneys, adrenal glands, and the urinary tract.

Legislation
Information and articles about legislation related to rare diseases, either bills in the U.S. Congress or laws enacted.

Metabolic Disorders
Biochemical disorders in which specific enzyme defects affect chemical processes in the body.

Mitochondrial disease
Diseases/disorders caused by damage to the mitochondria in the body's cells.

Movement disorders
Links to subject pages and feature articles on movement disorders (characterized by tics, tremors, chorea, and problems with balance and coordination).

Multiple Chemical Sensitivity
Illness caused by exposure to low levels of synthetic chemicals.

Multiple System Disorders
Diseases or disorders affecting more than one system of the body.

Muscles, Bones, and Joints
Disorders and diseases affecting the muscles, bones, and/or connective tissues in the body.

Muscular dystrophies
Muscular dystrophy is the overall name for a group of inherited muscle disorders that cause progressive weakening of the body's muscles. Find links here for information and resources for the different types.

Neurological Disorders
Disorders of the nervous system, including nerves, spinal cord, and brain.

Primary Immunodeficiency
An inherited problem with, or lack of, some or all of the body's immune system.

Prion Diseases
Diseases caused by prions (infectious proteins). These diseases include spongiform encephalopathies, kuru, Creutzfeldt-Jakob and Gerstmann-Straussler-Scheinker diseases, and fatal familial insomnia.

Purpura
Purple discolorations on the skin caused by bleeding underneath the skin. Small spots are petechiae, large spots are ecchymoses.

Respiratory Disorders
Rare disorders and diseases of the respiratory system.

Skin Disorders
Disorders, diseases, and conditions affecting the skin.

Storage diseases
Storage diseases are disorders in which some substance accumulates in cells in abnormal amounts. Find descriptions and links for Tay-Sachs, Gaucher, Fabry's, and other diseases on this page.

Lupus Erythematosus
A chronic inflammatory autoimmune connective tissue disease.

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