Bulletin, Spotlight

Five Things You Should Know – About Rare Diseases

  1. There are nearly 7,000 identified rare diseases, affecting 35 million Americans and more than 350 million individuals worldwide. If all of the people with a rare disease lived in one country, it would be the world’s 3rd most populous country.
  2. Approximately 50% of the people affected by rare diseases are children, and 35% of children with a rare disease will not live to see their 5th birthday.
  3. 95% of rare diseases do not have a single FDA-approved treatment or therapy.
  4. In 2016, Congress passed the 21st Century Cures Act, which included several provisions supported by CLSA to accelerate new therapies for rare diseases and ease the burden on families.
  5. To learn more about World Rare Disease Day, click here and for more information about rare diseases, check out NORD, EveryLife Foundation for Rare Diseases and Global Genes.