World Day for Rare Diseases
It is estimated that there are between 5,000 and 8,000 known rare diseases affecting 250 million people in the world. Shire has commercially approved and available treatments for Fabry disease, Hereditary Angioedema (HAE) and Hunter syndrome. In addition, Shire expects the U.S. Food & Drug Administration (FDA) to issue its decision for VPRIVTM (velaglucerase alfa for injection), Shire's investigational enzyme replacement treatment for Type 1 Gaucher disease, by February 28.
"For many patients and their families, the journey to a diagnosis and subsequent treatment can be a long, complicated process," said Sylvie Gregoire, President of Shire Human Genetic Therapies (HGT). "Shire is committed to enabling people with life-altering conditions to lead better lives - and for us, this includes improving access to information as well as providing much-needed treatment for rare diseases."