ea0095oc6.2 | Oral Communications 6 | BSPED2023
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...