Concepedia

Efficacy of Sapropterin Dihydrochloride in Increasing Phenylalanine Tolerance in Children with Phenylketonuria: A Phase III, Randomized, Double-Blind, Placebo-Controlled Study

Friedrich K. Trefz, Barbara K. Burton, Nicola Longo, Mercedes Martinez-Pardo Casanova, Daniel Gruskin, Alex Dorenbaum, Emil Kakkis, Eric Crombez, Dorothy K. Grange, Paul Harmatz,

The Journal of Pediatrics · 2009 · 192 citations · 30 references

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References

30

The metabolic and molecular bases of inherited disease

Ying-Tsong Chen · Medical Entomology and Zoology · 2001 · 6.9K citations

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Prefrontal Cortex Cognitive Deficits in Children Treated Early and Continuously for PKU

Adele Diamond, Meredith B. Prevor, Glenda G. Callender et al. · Monographs of the Society for Research in Child Development · 1997 · 722 citations

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Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study

Harvey L. Levy, Andrzej Milanowski, Anupam Chakrapani et al. · The Lancet · 2007 · 311 citations

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Tetrahydrobiopterin as an Alternative Treatment for Mild Phenylketonuria

Ania C. Muntau, Wulf Röschinger, Matthias Habich et al. · New England Journal of Medicine · 2002 · 305 citations · Full text

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How practical are recommendations for dietary control in phenylketonuria?

JH Walter, F. White, SK Hall et al. · The Lancet · 2002 · 301 citations

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