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...

ba0007oc14 | (1) | ICCBH2019

Burosumab resulted in greater improvement in clinical outcomes than continuation with conventional therapy in younger (1-4 years-old) and older (5-12 years-old) children with X-linked hypophosphatemia

Ward Leanne , Imel Erik , Whyte Michael , Munns Craig , Portale Anthony , Hogler Wolfgang , Simmons Jill , Padidela Raja , Namba Noriyuki , Cheong Hae , Nilsson Ola , Mao Meng , Skrinar Alison , Chen Chao-Yin , Martin Javier San , Glorieux Francis

Objective: We compared the efficacy and safety of burosumab, a monoclonal antibody against FGF23, to conventional therapy [oral phosphate and active vitamin D (Pi/D)] in children with X-linked hypophosphatemia (XLH).Methods: In this Phase 3 trial (NCT02915705), 61 children with XLH (1-12 years-old) were randomized 1:1 after a 7-day Pi/D washout to receive burosumab starting at 0.8 mg/kg SC Q2W or reinitiate Pi/D optimally titrated by investigators. Eligi...