Leading the future of rare disease medicine for patients like Cera.

We are committed to improving the lives of people with rare and ultra-rare genetic diseases through new medicines, educational initiatives, patient advocacy, research, and access to information.

Our diverse portfolio of approved medicines and investigational therapies is advancing first- or best-in-class treatments for diseases that exhibit a clear biology for treatment and are severely underserved by current standard of care.

We maintain an environment that puts people first. This includes patients, their families and caregivers, and our employees. We respect the responsibility that comes with developing medicines for rare diseases.