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What are the world's rarest diseases?

Treating Rare Diseases

Some countries require pharmaceutical companies to devote time and research to rare-disease treatments.
Some countries require pharmaceutical companies to devote time and research to rare-disease treatments. Schwartz

Humanity has struggled against disease for thousands of years, and it's a war not likely to end soon. Some of the more devastating epidemics in history either occurred in the last century, such as the Spanish flu, or continue to plague us today. Many of the victories in developing cures, vaccines and treatment programs come only after vast investments of time, money and research. Treating rare diseases is even more difficult.

If the efforts of global organizations and hundreds of scientists are still working to find cures and better treatments for epidemic diseases, such as AIDS, what chance do people with rare diseases have?


It may take time for doctors to accurately diagnose someone with a rare disease. However, once a diagnosis is made, there are a number of organizations that exist solely to help patients with rare diseases find the treatment they need. Additionally, some countries have legislation ensuring that rare diseases receive sufficient attention from researchers and pharmaceutical companies. This way, no matter how rare the disease, patients can expect to find treatment and the necessary orphan drugs, or medications developed to treat rare diseases. The U.S. Food and Drug Administration created the Office of Orphan Products Development to specifically devote resources for these types of diseases [source: FDA].

But does mandated research into rare conditions distract from finding new treatments and cures for widespread diseases? Not at all. The knowledge gained from studying rare diseases often has wide-reaching benefits. For instance, studies of the prion disease Kuru have led to a better understanding of similar, more widespread prion diseases, as well as the threat of mad cow disease.

To pay for the research and development, pharmaceutical companies often place high price tags, which many uninsured individuals cannot afford, on orphan drugs. Additionally, some rare diseases require treatment in a different state or country. This creates further financial hurdles, and many insurance programs place heavy restrictions on such treatment.

Advocacy groups such as the National Organization for Rare Disorders play an important role in ensuring patients have access to the treatments they need for full and productive lives.

To learn more about rare diseases and their advocacy and support groups and organizations, explore the links below.

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More Great Links


  • BBC News. "Rare condition named after twins." Oct. 13, 2005. (May 4, 2008)
  • Cann, Alan. "Prion Diseases." MicrobiologyBytes. Jan. 14, 2008. (May 4, 2008)
  • Centers for Disease Control and Prevention. "About Prion Diseases." Jan. 26, 2006. (May 4, 2008)
  • Centers for Disease Control and Prevention. "Smallpox Overview." Aug. 9, 2004. (May 3, 2008)
  • Janson, Paul A. "Kuru." Oct. 15, 2005. (May 4, 2008)
  • New York Times. "When a drug costs $300,000." March 23, 2008.
  • Nishikawa, Maya. "Insurance Struggle for Baby With Rare Disease." WCCO-TV. Oct. 20, 2007. (May 4, 2008)
  • Preston, Richard. "The Demon in the Freezer: Timeline." Random House. October 2002. (May 3, 2008)
  • Roach, John. "Cannibalism Normal for Early Humans?" National Geographic News. April 10, 2003. (May 4, 2008)
  • U.S. Food and Drug Administration. Office of Orphan Products Development. (May 6, 2008)
  • U.S. National Library of Medicine. "Rare Diseases." MedlinePlus. April 15, 2008. (May 4, 2008)
  • Watts, Joel C., et al. "The Expanding Universe of Prion Diseases." PloS Pathogens. 2006. (May 4, 2008)
  • Yount, Lisa. "Epidemics." Lucent Books. 2000.