Infinite potential.
One patient at a time.
Central to both our mission and vision is our commitment to the patients and families we serve.
A mission to transform
We are on a mission to transform the lives of patients with serious and rare diseases. We champion those who are overlooked and underserved and are dedicated to supporting patients along their journey. In everything we do, we put patients first – we talk with patients, listen to patients, and conduct our research with input from patients.
The vision to make it happen
Our vision is to be a globally recognized leading biotech company that empowers great people to deliver, with a profound sense of urgency and compassion, life-altering therapies to small patient populations experiencing big health problems.
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