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Currently, there are over 300 medicines under development to treat or prevent hundreds of rare diseases, as recently reported by the Pharmaceutical Research and Manufacturers of America (PhRMA).

The National Institutes of Health estimates that there are approximately 6,000 rare diseases affecting 25 million Americans. A rare disease is defined as a condition affecting fewer than 200,000 people in the United States.

Here are a few examples of medicines in development for rare diseases:

• A monoclonal antibody for chronic sarcoidosis, an immune system disorder.
• A medicine for Lennox-Gastaut syndrome, a severe form of epilepsy.
• Gene therapy for cystic fibrosis
• A medication for epidermolysis bullosa, a group of inherited disorders where skin blisters develop in response to minor trauma.
• A medicine for Friedreich's ataxia, a genetic disorder.

There are more than 160 medicines approved to treat rare diseases since 1995. The said report has been released by PhRMA together with Genetic Alliance and the National Organization for Rare Disorders (NORD).

Read the full press release.

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