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Tetrahydrobiopterin (BH4)

BH4 loading test, disorders of BH4 synthesis, BH4 responsive PKU. Currently, replaced by Kuvan®.

BH4 loading test: 20 mg/kg/dose once daily for 2 consecutive days.

Others: 5-20 mg/kg/day, monitor phenylalanine levels and adjust the dose accordingly (160, 167).

50 mg tablet

PO

Abdominal pain, diarrhea, nausea, vomiting, headache, nasal discharge, throat pain, URTI.

4

160. Brandt NJ, Gregersen N, Christensen E, Gron IH, Rasmussen K. Treatment of glutaryl-CoA dehydrogenase deficiency (glutaric aciduria). Experience with diet, riboflavin, and GABA analogue. J Pediatr 1979;94(4):669-73

167. Levy HL, Milanowski A, Chakrapani A, et al. Efficacy of sapropterin dihydrochloride (tetrahydrobiopterin, 6R-BH4) for reduction of phenylalanine concentration in patients with phenylketonuria: a phase III randomised placebo-controlled study. Lancet 2007;370(9586):504-10 doi: 10.1016/S0140-6736(07)61234-3[published Online First: Epub Date]|.

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