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Diagnostic and therapeutic recommendations for the treatment of hyperphenylalaninemia in patients 0–4 years of age
BACKGROUND: Treatment of phenylketonuria (PKU) with sapropterin dihydrochloride in responsive patients from an early age can have many advantages for the patient over dietary restriction alone. Accordingly, approval of sapropterin in the European Union was extended in 2015 to include patients aged 0...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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BioMed Central
2018
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6162894/ https://www.ncbi.nlm.nih.gov/pubmed/30268140 http://dx.doi.org/10.1186/s13023-018-0911-6 |