A Japanese single-center experience of the efficacy and safety of asfotase alfa in pediatric-onset hypophosphatasia

BACKGROUND: Hypophosphatasia (HPP) is a rare inherited metabolic disorder caused by mutations in the ALPL gene, which encodes tissue nonspecific alkaline phosphatase. The severity of HPP is widely diverse from the perinatal form to the adult mild form. The former represents the most severe form and...

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Detalles Bibliográficos
Autores principales: Sugiyama, Yohei, Watanabe, Taijiro, Tajika, Makiko, Matsuhashi, Tetsuro, Shimura, Masaru, Fushimi, Takuya, Ichimoto, Keiko, Matsunaga, Ayako, Ebihara, Tomohiro, Tsuruoka, Tomoko, Akiyama, Tomoyuki, Murayama, Kei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8867653/
https://www.ncbi.nlm.nih.gov/pubmed/35197081
http://dx.doi.org/10.1186/s13023-022-02230-y

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