Alexion is accelerating the development of asfotase alfa , a first-in-class targeted enzyme replacement therapy in Phase 2 clinical trials for Hypophosphatasia (HPP). HPP is an ultra-rare, genetic and life-threatening metabolic disease with no effective treatment options. Read more
On Feb. 29, Alexion joins patient groups, families, governments and medical communities worldwide to celebrate Rare Disease Day and raise awareness of the unique challenges families face in living with these disorders.
When Victor Estevez developed PNH, the upbeat, energetic event worker who was busy planning a future with his wife-to-be was suddenly facing long hospitalizations, debilitating pain and fatigue, and a sense of isolation from his loved ones. Find out how Victor learned to manage his disease and is once again sharing happy moments with his growing family. Play video.