Searchable abstracts of presentations at key conferences on calcified tissues

ba0003pp363 | Other diseases of bone and mineral metabolism | ECTS2014

Sustained efficacy and tolerability in infants and young children with life-threatening hypophosphatasia treated with asfotase alfa

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...