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

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 with pe...

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Detalles Bibliográficos
Autores principales: Dahir, Kathryn M., Seefried, Lothar, Kishnani, Priya S., Petryk, Anna, Högler, Wolfgang, Linglart, Agnès, Martos-Moreno, Gabriel Ángel, Ozono, Keiichi, Fang, Shona, Rockman-Greenberg, Cheryl
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9295501/
https://www.ncbi.nlm.nih.gov/pubmed/35854311
http://dx.doi.org/10.1186/s13023-022-02393-8