Impact
We are driven to make an impact for patients and their caregivers—from early detection to therapeutic intervention.
Our Guiding Principles
Our goal is to provide novel biopharmaceutical medicines that extend, improve, or enhance the lives of people with rare diseases. We will adhere to these foundational principles:
Innovation
We are committed to providing differentiated, high-quality solutions that address unmet medical needs for those who suffer from rare diseases.
Integrity
We will never compromise our integrity and will lead with transparency, accountability, and compassion.
