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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.
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).
