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CAS 62989-33-7

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Sapropterin

Description:
Sapropterin, also known as 6R-L-erythro-5,6,7,8-tetrahydrobiopterin, is a synthetic form of the naturally occurring cofactor tetrahydrobiopterin (BH4). It is primarily used in the treatment of phenylketonuria (PKU), a genetic disorder that leads to the accumulation of phenylalanine due to a deficiency in the enzyme phenylalanine hydroxylase. Sapropterin acts by enhancing the activity of this enzyme, thereby facilitating the metabolism of phenylalanine. The substance is characterized by its ability to cross the blood-brain barrier, which is crucial for its therapeutic effects. In terms of its chemical structure, sapropterin contains a pteridine ring, which is essential for its biological activity. It is typically administered orally and is well-tolerated, although some patients may experience mild side effects. The compound is classified as a pharmaceutical agent and is subject to regulatory oversight to ensure its safety and efficacy in clinical use. Overall, sapropterin represents a significant advancement in the management of PKU, allowing for better dietary control and improved quality of life for affected individuals.
Formula:C9H15N5O3
InChI:InChI=1S/C9H15N5O3/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)/t3-,4+,6-/m0/s1
InChI key:InChIKey=FNKQXYHWGSIFBK-RPDRRWSUSA-N
SMILES:O=C1C2=C(NC[C@]([C@H]([C@H](C)O)O)(N2)[H])NC(N)=N1
Synonyms:
  • (6R)-2-Amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-4(1H)-pteridinone
  • (6R)-2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydropteridin-4(1H)-one
  • (6R)-5,6,7,8-Tetrahydro-<span class="text-smallcaps">L</span>-biopterin
  • (6R)-5,6,7,8-Tetrahydrobiopterin
  • (6R)-<span class="text-smallcaps">L</span>-erythro-5,6,7,8-Tetrahydrobiopterin
  • (6R)-<span class="text-smallcaps">L</span>-erythro-Tetrahydrobiopterin
  • (6R)-Tetrahydrobiopterin
  • (6R,S)-5,6,7,8-Tetrahydro-L-Biopterin Dihydrochloride
  • 2-amino-6-(1,2-dihydroxypropyl)-5,6,7,8-tetrahydropteridin-4(1H)-one
  • 2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydropteridin-4(1H)-one
  • See more synonyms
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Found 3 products.