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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...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2022
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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 |