Searchable abstracts of presentations at key conferences in endocrinology

ea0095oc6.2 | Oral Communications 6 | BSPED2023

Children with hypophosphatasia treated with asfotase alfa: analysis from the UK Patients Cohort

Padidela Raja , Bishop Nick , Arundel Paul , Fang Shona , Zygouras Alexandros , Mughal Zulf , Shaw Nick , Saraff Vrinda

Objective: To describe outcomes among children with hypophosphatasia (HPP) receiving asfotase alfa.Methods: This prospective, real-world study used data from all children with HPP receiving asfotase alfa in the UK Managed Access Agreement (MAA) to assess functional, health-related quality-of-life, and safety outcomes. Visits occurred at MAA enrolment, 3 and 6 months after enrolment, and every 6 months thereafter. Assessm...