Clinical profiles of treated and untreated adults with hypophosphatasia in the Global HPP Registry

Clinical profiles of treated and untreated adults with hypophosphatasia in the Global HPP Registry

Abstract Background The clinical signs and symptoms of hypophosphatasia (HPP) can manifest during any stage of life. The age at which a patient’s symptoms are reported can impact access to targeted treatment with enzyme replacement therapy (asfotase alfa), as this treatment is indicated for patients...

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Bibliographic Details
Main Authors: Kathryn M. Dahir, Lothar Seefried, Priya S. Kishnani, Anna Petryk, Wolfgang Högler, Agnès Linglart, Gabriel Ángel Martos-Moreno, Keiichi Ozono, Shona Fang, Cheryl Rockman-Greenberg
Format: Article
Language:English
Published: BMC 2022-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Hypophosphatasia
Burden of disease
Enzyme replacement therapy
HRQoL
Online Access:https://doi.org/10.1186/s13023-022-02393-8

Internet

https://doi.org/10.1186/s13023-022-02393-8

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