Alexion Pharmaceuticals Inc. (ALXN) Release: New Data Detailing The Devastating Nature Of Hypophosphatasia (HPP) And Treatment Effect Of Asfotase Alfa In Infants, Young Children And Juveniles With HPP Presented At The Pediatric Academic Societies Meeting
5/6/2014 8:25:50 AM
CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (Nasdaq:ALXN) today announced that researchers have presented new data from a large, retrospective, natural history study of patients with severe perinatal and infantile hypophosphatasia (HPP), an inherited, rare metabolic disorder that can lead to progressive damage to multiple vital organs, destruction and deformity of bones, and death. The natural history study details the devastating nature of the disease, with 73% mortality reported at five years. Additionally, early and sustained improvements were observed in infants, children and juveniles receiving asfotase alfa, an investigational therapy for the treatment of HPP, in the open-label extension phase of two on-going Phase 2 clinical studies. The data were presented in poster sessions at the joint meeting of the Pediatric Academic Societies (PAS) and the Asian Society for Pediatric Research in Vancouver, B.C., Canada.
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