Tag Archives: rare disease

Rare diseases gain needed attention

iStock_000008847188XSmall“Rare diseases” are those that affect less than 200,000 people in the United States. Because these diseases aren’t as common, recruitment for clinical trials tends to be difficult, and patients are usually limited in treatment options. But as there are currently over 7,000 diseases that fall into this category, one in ten Americans is affected, making rare diseases pretty significant.

Pharmaceutical companies to the rescue! Currently, 452 new medicines and vaccines for rare diseases are in development- either currently in human clinical trials or under FDA review. Included in these new drugs are drugs that will be useful for genetic disorders, infectious diseases, Lou Gehrig’s disease, leukemia, and other types of cancer- including pediatric cancers.

As many rare diseases affect children, this is good news for everyone! As a bonus, researchers find that understanding rare diseases can often provide insight into more common disorders. Biomedical research to the rescue…