First Japanese case of maternal phenylketonuria treated with sapropterin dihydrochloride and the normal growth and development of the child
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
Reference16 articles.
1. Maternal phenylketonuria and hyperphenylalaninemia. An international survey of the outcome of untreated and treated pregnancies;Lenke;N. Engl. J. Med.,1980
2. Use of sapropterin dihydrochloride in maternal phenylketonuria. A European experience of eight cases;Feillet;J. Inherit. Metab. Dis.,2014
3. Barriers to successful dietary control among pregnant women with phenylketonuria;Brown;Genet Med.,2002
4. Identification of pharmacological chaperones as potential therapeutic agents to treat phenylketonuria;Pey;J. Clin. Invest.,2008
5. Tetrahydrobiopterin and maternal PKU;Koch;Mol. Genet. Metab.,2005
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1. Cell Therapy Development for Congenital Metabolic DiseasesUsing Human Amniotic Epithelial Cells;Journal of Nihon University Medical Association;2023-06-01
2. Sapropterin for phenylketonuria: A Japanese post‐marketing surveillance study;Pediatrics International;2022-01
3. Guide for diagnosis and treatment of hyperphenylalaninemia;Pediatrics International;2021-01
4. Similarities and differences in key diagnosis, treatment, and management approaches for PAH deficiency in the United States and Europe;Orphanet Journal of Rare Diseases;2020-09-25
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