The drive to become a leading biotech company focused on innovative approaches to treat rare and specialty diseases, shapes the foundation of our Research and Development approach. Our expertise in discovering new therapies for rare, life-threatening genetic diseases as well as in developing medicines for patients treated by specialist physicians, demonstrates our continued commitment to patients and their caregivers.
Our R&D Strategy focuses on rare and specialty diseases with high unmet medical needs.
We strive for operational excellence in a wide range of clinical studies.
We are committed to supporting scientific research and education.