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Sapropterin ((6R)-BH4) dihydrochloride is a synthetic form of BH4 that is approved for the treatment of BH4 responsive PKU. (1) Sapropterin dihydrochloride can stimulate TH and TPH activities leading to improved dopamine and serotonin synthesis despite persistently elevated brain phenylalanine. (2) Sapropterin dihydrochloride is used to lower blood phenylalanine levels in tetrahydrobiopterin-responsive phenylketonuria in conjunction with a phenylalanine-restricted diet.
| Molecular Weight | 314.17 |
| CAS Number | 69056-38-8 |
| Solubility (25°C) | Water 90 mg/mL |
| Storage | 2-8°C, dry, sealed |
[1] No authors listed. Sapropterin
[2] Usha Rani Somaraju, et al. Sapropterin dihydrochloride for phenylketonuria
[3] Nenad Blau. Sapropterin dihydrochloride for the treatment of hyperphenylalaninemias
[4] Usha Rani Somaraju, et al. Sapropterin dihydrochloride for phenylketonuria
[5] Usha Rani Somaraju, et al. Sapropterin dihydrochloride for phenylketonuria
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