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Introduction of sapropterin dihydrochloride as standard of care in patients with phenylketonuria
Sapropterin dihydrochloride, a synthetic, stable form of the tetrahydrobiopterin cofactor of phenylalanine hydroxylase, has been shown to reduce plasma phenylalanine (Phe) levels in a significant portion of patients with phenylketonuria (PKU). When we undertook introducing this medication to our PKU...
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| Autori principali: | , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2010
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3135651/ https://ncbi.nlm.nih.gov/pubmed/20418136 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2010.03.022 |
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