Open AccessEfficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study
Author(s) -
Christine Hofmann,
Paul Harmatz,
Jerry Vockley,
Wolfgang Högler,
Hideki Nakayama,
Nick Bishop,
Gabriel Ángel MartosMoreno,
Scott Moseley,
Kenji P. Fujita,
Johannes G. Liese,
Cheryl R. Greenberg,
ENB-- Study Group
Publication year - 2019
Publication title -
the journal of clinical endocrinology and metabolism
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.206
H-Index - 353
eISSN - 1945-7197
pISSN - 0021-972X
DOI - 10.1210/jc.2018-02335
Subject(s) - hypophosphatasia , open label , medicine , pediatrics , pharmacology , adverse effect , alkaline phosphatase , chemistry , biochemistry , enzyme
Long-term data on enzyme replacement treatment of hypophosphatasia (HPP) are limited.
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