How do you define a rare disease?

In the United States, a rare disease is defined as a condition that affects fewer than 200,000 people in the US. This definition was created by Congress in the Orphan Drug Act of 1983. Rare diseases became known as orphan diseases because drug companies were not interested in adopting them to develop treatments.

Which diseases can be classified as rare diseases?

Rare Disease Database

• 47,XXY (Klinefelter Syndrome) Also known as: KS, XXY male.
• Aarskog Syndrome.
• Abetalipoproteinemia.
• Ablepharon-Macrostomia Syndrome.
• Acanthocheilonemiasis.
• Acanthosis Nigricans.
• Aceruloplasminemia.
• Achalasia.

What is the definition of a rare disease?

Definition. In the United States, the Rare Diseases Act of 2002 defines rare disease strictly according to prevalence, specifically “any disease or condition that affects fewer than 200,000 people in the United States”, or about 1 in 1,500 people. This definition is essentially the same as that of the Orphan Drug Act of 1983,…

Are there any diseases that are not inherited?

Many rare diseases, including infections, some rare cancers, and some autoimmune diseases, are not inherited. While researchers are learning more each year, the exact cause of many rare diseases is still unknown.

How many people are affected by Rare Diseases in Europe?

Although this might appear small, it translates into approximately 246,000 people. Most patients suffer from even rarer diseases affecting 1 person in 100,000 or more. Approximately 5,000-8,000 distinct rare diseases affect 6-8% of the EU population i.e. between 27 and 36 million people. What is the EU doing?

Are there any treatments or cures for rare diseases?

Researchers have made progress in learning how to diagnose, treat, and even prevent a variety of rare diseases. However, there is still much to do because most rare diseases have no treatments. The National Institutes of Health (NIH) supports research to improve the health of people with rare diseases.