We are a global biotechnology company at the forefront of advancing therapies for a range of rare and devastating diseases and our excellent team has a very big and a very bold vision.

Each program that we invest in has to have three key characteristics. It has to be in the rare diseases for a devastating disorder, the technology has to have the potential to be first in class or best in class, and the clinical data must be able to provide meaningful benefit for patients. As we pursue treatments for rare disorders, we maintain a personal and compassionate focus on patients, their caregivers, and families.

This personal and direct relationship with patients embodies the culture of our organization and drives what we do very passionately every day. Every biotech company says it’s focused on patients, but at Amicus we do it in a different way. You see it when you go to our offices around the world where we have pictures of patients with the diseases we treat accompanied by narratives about their struggles, identities, and lives.

How do we take that perspective and build it into every part of the business? All the way from team meetings to the board of directors, we try to make all our decisions as if we were living with these diseases or were a mom or dad or kid caring for someone with this disorder. What would be important to me as a patient? What would be a meaningful advancement or a risk I would take? We do more than say that we are patient focused – we live it.