Physical Function and Health‐Related Quality of Life in Adults Treated With Asfotase Alfa for Pediatric‐Onset Hypophosphatasia

Hypophosphatasia (HPP) is a rare, inherited, metabolic disease characterized by tissue‐nonspecific alkaline phosphatase deficiency resulting in musculoskeletal and systemic clinical manifestations. This observational study evaluated the effectiveness of enzyme replacement therapy with asfotase alfa...

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Detalles Bibliográficos
Autores principales: Genest, Franca, Rak, Dominik, Petryk, Anna, Seefried, Lothar
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2020
Materias:
Clinical Trial
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7507107/
https://www.ncbi.nlm.nih.gov/pubmed/32995696
http://dx.doi.org/10.1002/jbm4.10395

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7507107/
https://www.ncbi.nlm.nih.gov/pubmed/32995696
http://dx.doi.org/10.1002/jbm4.10395

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