Rare Humanitarian Program

Our Rare Humanitarian Program, the first and longest running humanitarian initiative of its kind, for people with lysosomal storage disorders, began in 1991 when our first treatment for Gaucher disease was approved by the U.S. FDA. For more than 30 years, the program has been focused on providing access to free treatments for people with rare diseases meeting the program’s criteria, who otherwise would not have access to such treatments.

Through this interactive experience, explore first-hand stories from patients, families, physicians and partners. Their personal journeys of strength, determination, perseverance and hope help to define this program and inspire us to do more.