Hypophosphatasia: Enzyme Replacement Therapy (Asfotase Alfa) Decreases TNSALP Substrate Accumulation And Improves Functional Outcomes In Affected Adolescents And Adults

1Shriners Hospital for Children, St. Louis, MO USA; 2Duke University, Durham, NC; 3University of Manitoba, Winnipeg, Manitoba, Canada; 4Shriners Hospital, St. Louis, MO; 5University of Manitoba, Winnipeg, Manitoba, Canada; 6Alexion Pharmaceuticals, Cambridge, MA; 7Corresponding Author: Nerissa Kreher, MD, Alexion Pharmaceuticals, 55 Cambridge Parkway, Ste 800, Cambridge,MA 02142 USA, 617-674-5739; email:Nerissa.Kreher@alxn.com Whyte MP1, Kishnani PS2, Greenberg CR3, Madson K4, Mack K4, Weber T2, Mhanni A5, Plotkin H6, Kreher N6, Landy H6, Nerissa Kreher7 SHORT COMMUNICATION