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

Summary Background Early and strict dietary management of phenylketonuria is the only option to prevent mental retardation. We aimed to test the efficacy of sapropterin, a synthetic form of tetrahydrobiopterin (BH4), for reduction of blood phenylalanine concentration. Methods We enrolled 89 patients...

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Veröffentlicht in:	The Lancet (British edition) 2007-08, Vol.370 (9586), p.504-510
Hauptverfasser:	Levy, Harvey L, Dr, Milanowski, Andrzej, MD, Chakrapani, Anupam, MD, Cleary, Maureen, MD, Lee, Philip, MD, Trefz, Friedrich K, MD, Whitley, Chester B, MD, Feillet, François, MD, Feigenbaum, Annette S, FRCPC, Bebchuk, Judith D, ScD, Christ-Schmidt, Heidi, MSE, Dorenbaum, Alex, MD
Format:	Artikel
Sprache:	eng
Schlagworte:	Adolescent Adult Biopterins - adverse effects Biopterins - analogs & derivatives Biopterins - therapeutic use Blood Child Children & youth Clinical trials Double-Blind Method Female Hospitals Humans Internal Medicine Male Middle Aged Phenylalanine - blood Phenylketonurias - blood Phenylketonurias - drug therapy Placebo effect Respiratory tract Treatment Outcome Womens health
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