At Shire, we are champions for people around the world who are struggling with rare diseases – they are at the center of everything we do.
Focusing on a Significant Unmet Medical Need
Rare diseases, most of which are genetic and are present throughout a person's entire life, pose a significant medical and economic burden for patients, communities and healthcare systems. There are more than 7,000 known rare diseases, including Hemophilia, Hereditary Angioedema (HAE), Gaucher disease, Hunter Syndrome, and many forms of rare cancer, and an estimated 350 million people worldwide are living with one.1
These figures do not reflect the untold number of family members and friends who watch a loved one struggle with health challenges that, in many cases, cannot be adequately addressed today, especially considering nearly half of the time these loved ones are children. What's more, patients with rare diseases commonly experience delays to diagnosis, which can lead to serious consequences for their health and the wider healthcare system.