Chaperone-Like Therapy with Tetrahydrobiopterin in Clinical Trials for Phenylketonuria: Is Genotype a Predictor of Response?

Prospectively enrolled phenylketonuria patients (n=485) participated in an international Phase II clinical trial to identify the prevalence of a therapeutic response to daily doses of sapropterin dihydrochloride (sapropterin, KUVAN(®)). Responsive patients were then enrolled in two subsequent Phase...

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Hauptverfasser: Sarkissian, Christineh N., Gamez, Alejandra, Scott, Patrick, Dauvillier, Jerome, Dorenbaum, Alejandro, Scriver, Charles R., Stevens, Raymond C.
Format: Artigo
Sprache:Inglês
Veröffentlicht: Springer Berlin Heidelberg 2011
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3509924/
https://ncbi.nlm.nih.gov/pubmed/23430918
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2011_96
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