Our Commitment to Patients

We believe the long-term value of innovation in healthcare is critical for the many patients with rare diseases who are still waiting for an approved treatment. We therefore dedicate ourselves every day to the goal of providing treatments where none currently exist. This commitment is based on the fundamental principles of advancing innovative life-changing treatments as quickly as possible and effectively providing these treatments to patients in need. We aim to develop safe and effective treatments for many serious rare diseases as fast as we can, and we are committed to helping the whole rare disease community move forward by sharing our science and expertise to advance future development, whether by us or others.