Sapropterin for phenylketonuria: A Japanese post‐marketing surveillance study

BACKGROUND: The aim of this study was to assess the long‐term safety and efficacy of sapropterin in a real‐world setting in Japanese patients with tetrahydrobiopterin (BH4)‐responsive phenylketonuria. METHODS: This post‐marketing surveillance study enrolled all of the patients in Japan with confirme...

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Detalles Bibliográficos
Autores principales: Tamura, Mina, Seki, Shizuka, Kakurai, Yasuyuki, Chikada, Shuichi, Wada, Kento
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2022
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9305189/
https://www.ncbi.nlm.nih.gov/pubmed/34331785
http://dx.doi.org/10.1111/ped.14939

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9305189/
https://www.ncbi.nlm.nih.gov/pubmed/34331785
http://dx.doi.org/10.1111/ped.14939

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