Effectiveness of asfotase alfa for treatment of adults with hypophosphatasia: results from a global registry.

Priya S KishnaniGabriel Ángel Martos-MorenoAgnès LinglartAnna PetrykAndrew MessaliShona FangCheryl Rockman-GreenbergKeiichi OzonoWolfgang HöglerLothar SeefriedKathryn M Dahir

Published in: Orphanet journal of rare diseases (2024)

NCT02306720; EUPAS13514.

Keyphrases

replacement therapy
randomized controlled trial
systematic review