ba0003pp363 | Other diseases of bone and mineral metabolism | ECTS2014
Whyte Michael
, Simmons Jill
, Lutz Richard
, Vallee Marc
, Melian Agustin
, Odrljin Tatjana
, Bishop Nick
Background: Hypophosphatasia (HPP) results from inactivating mutation(s) in the gene for tissue non-specific alkaline phosphatase (TNSALP). Substantial improvement has been reported in skeletal mineralization and physical function in patients (pts) with life-threatening perinatal and infantile HPP treated for 48 weaks with asfotase alfa, a bone-targeted recombinant human TNSALP.Objective: To evaluate long-term efficacy and tolerability of asfotase alfa i...