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CAS 69056-38-8

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Sapropterin dihydrochloride

Description:
Sapropterin dihydrochloride is a synthetic form of the naturally occurring cofactor tetrahydrobiopterin (BH4), primarily used in the treatment of phenylketonuria (PKU), a metabolic disorder characterized by the inability to metabolize phenylalanine. This compound acts as a pharmacological chaperone, enhancing the activity of phenylalanine hydroxylase, the enzyme responsible for converting phenylalanine to tyrosine. Sapropterin dihydrochloride is a white to off-white crystalline powder that is soluble in water, making it suitable for oral administration. Its chemical formula reflects its dihydrochloride salt form, which contributes to its stability and solubility. The substance is generally well-tolerated, with common side effects including headache, gastrointestinal disturbances, and allergic reactions. As a therapeutic agent, it plays a crucial role in managing blood phenylalanine levels, thereby preventing the neurological complications associated with PKU. Its use is typically monitored through regular blood tests to ensure efficacy and safety in patients.
Formula:C9H15N5O3·2ClH
InChI:InChI=1S/C9H15N5O3.2ClH/c1-3(15)6(16)4-2-11-7-5(12-4)8(17)14-9(10)13-7;;/h3-4,6,12,15-16H,2H2,1H3,(H4,10,11,13,14,17);2*1H/t3-,4+,6-;;/m0../s1
InChI key:InChIKey=RKSUYBCOVNCALL-NTVURLEBSA-N
SMILES:O=C1C2=C(NC[C@]([C@H]([C@H](C)O)O)(N2)[H])NC(N)=N1.Cl
Synonyms:
  • (6R)-2-Amino-6-((1R,2S)-1,2-dihydroxypropyl)-5,6,7,8-tetrahydro-4(1H)-pteridinone dihydrochloride
  • (6R)-2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydropteridin-4(1H)-one dihydrochloride
  • (6R)-5,6,7,8-Tetrahydro-L-Biopterin Dihydrochloride
  • (6R)-Tetrahydrobiopterin hydrochloride
  • (6β)-5,6,7,8-Tetrahydro-<span class="text-smallcaps">L</span>-biopterin dihydrochloride
  • 2-amino-6-[(2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydropteridin-4(1H)-one dihydrochloride
  • 4(1H)-Pteridinone, 2-amino-6-(1,2-dihydroxypropyl)-5,6,7,8-tetrahydro-, dihydrochloride, [6R-[6R*(1R*,2S*)]]-
  • 4(1H)-Pteridinone, 2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-, dihydrochloride, (6R)-
  • 4(1H)-Pteridinone, 5,6,7,8-tetrahydro-2-amino-6-(1,2-dihydroxypropyl)-, dihydrochloride, (6R-(6R*(1R*,2S*)))-
  • 4(3H)-Pteridinone, 2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-, hydrochloride (1:2), (6R)-
  • See more synonyms
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Purity (%)
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100
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0
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50
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90
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95
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100
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