WebMay 17, 2005 · Both products have shown potential in the treatment of phenylketonuria (PKU), and there is preliminary clinical evidence that suggests that the active ingredient in Phenoptin, a synthetic form of the naturally occurring enzyme cofactor 6R-BH4, may also be useful in the treatment of other serious diseases, including diabetes and cardiovascular ... WebMar 16, 2006 · Phenoptin is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Phenoptin, sapropterin dihydrochloride, is the synthetic form of 6R-BH4 ( tetrahydrobiopterin), a naturally occurring enzyme cofactor that works in conjunction with phenylalanine hydroxylase (PAH) to metabolize Phe.
Merck Serono/BioMarin
WebJan 26, 2006 · Phenoptin is an investigational oral small molecule therapeutic for the treatment of PKU. The active ingredient in Phenoptin, sapropterin dihydrochloride, is the synthetic form of 6R-BH4, a naturally occurring enzyme cofactor. BioMarin received orphan drug designation for Phenoptin to treat PKU from both the FDA and European Medicines … WebJun 14, 2008 · Treatments consisted of 3 consecutive 2-week courses of Kuvan at doses of 5, then 20, and then 10 mg/kg/day. Blood Phe level was monitored after 2 weeks of treatment at each dose level. At baseline, mean (±SD) blood Phe was 844 (±398) µmol/L. cshcn paf form
Sapropterin dihydrochloride (Kuvan/phenoptin), an orally …
WebJul 19, 2006 · The purpose of this study is to evaluate the ability of Phenoptin to control blood phenylalanine levels in subjects who have hyperphenylalaninemia due to a prim... English Dansk Deutsch English Español Français Italiano Magyar Nederlands Norsk Polski Português Suomi Svenska Čeština Русский 日本語 简体中文 한국어 WebDec 19, 2006 · Data demonstrated the long-term safety and tolerability of Phenoptin as a treatment to control blood Phe levels across a range of doses in PKU patients. The 22-week multi-center open-label dose titration Phase 3 extension study enrolled 80 patients who had previously completed the pivotal Phase 3 clinical trial. Patients in the extension study ... eagan mall stores