Our difference is that we make a difference.
We focus on rare genetic disorders with high unmet medical need. Whether it be providing communities with their first treatment option, or improving upon the current standard of care, we focus on conditions where we believe we can make a meaningful impact.
Our Products
We find tomorrows.
Each of our approved therapies was driven by the goal of resetting the standard of care for the conditions they treat.
Learn More about our products ⌃Patient Stories
Patients inspire and enable our work every day. Meet some of the exceptional people whose stories remain at the heart of everything we do.
Learn More about our patients ⌃Our Pipeline
Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition it will address.
Learn More about our pipline ⌃