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Research About Rare Diseases

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Information about research into rare diseases, including summaries of research studies, genetics, clinical trials, and orphan drugs.

  1. Clinical Trials
  2. Orphan Drugs

Clinical Trials

Information about clinical trials for rare diseases -- what they are, how to enroll, and which ones are looking for volunteers.

Orphan Drugs

"Orphan drug" is the name given to a drug developed to treat a rare disease. Find information here about orphan drugs, including what they are and which ones have been approved in the U.S. and Europe.

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