Long-term preservation of intellectual functioning in sapropterin-treated infants and young children with phenylketonuria: A seven-year analysis

Sapropterin dihydrochloride has been approved for the treatment of hyperphenylalaninemia in infants and young children with phenylketonuria (PKU). Sapropterin can reduce phenylalanine (Phe) levels in tetrahydrobiopterin (BH4)-responsive patients, potentially preventing the intellectual impairment ca...

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Detalles Bibliográficos
Autores principales: Waisbren, Susan, Burton, Barbara K., Feigenbaum, Annette, Konczal, Laura L., Lilienstein, Joshua, McCandless, Shawn E., Rowell, Richard, Sanchez-Valle, Amarilis, Whitehall, Kaleigh B., Longo, Nicola
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8684368/
https://www.ncbi.nlm.nih.gov/pubmed/33485801
http://dx.doi.org/10.1016/j.ymgme.2021.01.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8684368/
https://www.ncbi.nlm.nih.gov/pubmed/33485801
http://dx.doi.org/10.1016/j.ymgme.2021.01.001

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