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Oct 20, 2008
 |  BC Week In Review  |  Clinical News  |  Clinical Results

6R-BH4 sapropterin dihydrochloride: Phase IIa data

In a 16-week, dose-escalation, open-label, U.S. Phase IIa study in 32 patients with SCD, 6R-BH4 showed a dose-dependant improvement in endothelial dysfunction with the mean PAT score for all patients improving from 1.58 at baseline to 1.90 at week 16. Patients received 2.5, 5,...

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