ABOUT US
Every Day We Change Lives
People living with rare and devastating diseases are our Guiding Star. We believe it is our responsibility to listen to, understand, and change the lives of patients and those who work tirelessly to help them.
Our Mission
To transform the lives of people affected by rare diseases and devastating conditions by continuously innovating and creating meaningful value in all that we do.
And we’re making real progress, every day.
Every Day
Strengthens Our
Impact
We continue to deepen our understanding of rare disease, which began with our pioneering work in complement biology. This knowledge allows us to innovate and evolve into new areas, where there is great unmet need and opportunity to help patients and families fully live their best lives.
We’ve delivered transformative medicines for people with paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), anti-aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD), generalized Myasthenia Gravis (gMG), hypophosphatasia (HPP), and lysosomal acid lipase deficiency (LAL-D).
