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Impact of discontinuing 5 years of enzyme replacement treatment in a cohort of 6 adults with hypophosphatasia: A case series

Asfotase alfa is a human recombinant enzyme replacement therapy for hypophosphatasia. We describe 6 adults who were treated with asfotase alfa for 61–68 months in a clinical trial (NCT01163149), after which asfotase alfa was discontinued for 15–48 months. The patients experienced clinical deteriorat...

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Detalles Bibliográficos
Autores principales: Rockman-Greenberg, Cheryl, Josse, Robert, Francis, Mira, Mhanni, Aziz
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9463174/
https://www.ncbi.nlm.nih.gov/pubmed/36097602
http://dx.doi.org/10.1016/j.bonr.2022.101617