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
Alexion was honored by the National Organization for Rare Disorders (NORD) with a “Partners in Progress” Corporate Award for bringing to market the first and only treatment for patients with atypical hemolytic uremic syndrome (aHUS).
The award was given at the NORD gala in Washington, D.C. on May 15th, an annual event that honors government officials, patient advocates, medical researchers, and companies that have made a difference in the lives of patients with rare diseases.
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.