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Sapropterin dihydrochloride

Cat. No. M14981
Sapropterin dihydrochloride Structure
Synonym:

(6R)-BH4 dihydrochloride; (6R)-Tetrahydro-L-biopterin dihydrochloride

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Quality Control & Documentation
Biological Activity

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.

Chemical Information
Molecular Weight 314.17
CAS Number 69056-38-8
Solubility (25°C) Water 90 mg/mL
Storage 2-8°C, dry, sealed
Conversion of different model animals based on BSA (PMID: 27057123)
Species Mouse Rat Rabbit Guinea pig Hamster Dog
Weight (kg) 0.02 0.15 1.8 0.4 0.08 10
Body Surface Area (m2) 0.007 0.025 0.15 0.05 0.02 0.5
Km factor 3 6 12 8 5 20
Animal A (mg/kg) = Animal B (mg/kg) multiplied by  Animal B Km
Animal A Km

For example, to modify the dose of Compound A used for a mouse (20 mg/kg) to a dose based on the BSA for a rat, multiply 20 mg/kg by the Km factor for a mouse and then divide by the Km factor for a rat. This calculation results in a rat equivalent dose for Compound A of 10 mg/kg.

References

[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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Keywords: Sapropterin dihydrochloride, (6R)-BH4 dihydrochloride; (6R)-Tetrahydro-L-biopterin dihydrochloride supplier, Others, inhibitors, activators


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