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Brand names,
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Analogs
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Brand Names Mixture
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Chemical_Formula
C9H15N5O3
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RX_link
No information avaliable
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fda sheet
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msds (material safety sheet)
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Synthesis Reference
No information avaliable
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Molecular Weight
241.247 g/mol
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Melting Point
250-255 oC (hydrochloride salt)
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H2O Solubility
>20 mg/mL (dichloride salt)
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State
Solid
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LogP
-1.783
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Dosage Forms
No information avaliable
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Indication
For the treatment of tetrahydrobiopterin (BH4) deficiency.
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Pharmacology
Tetrahydrobiopterin (BH4) is used to convert several amino acids, including phenylalanine, to other essential molecules in the body including neurotransmitters. Tetrahydrobiopterin deficiency can be caused by mutations in GTP cyclohydrolase 1 (GCH1), 6-pyruvoyl-tetrahydropterin synthase/dimerization cofactor of hepatocyte nuclear factor 1 alpha (PCBD1), 6-pyruvoyltetrahydropterin synthase (PTS), and quinoid dihydropteridine reductase (QDPR) genes. These genes make the enzymes that are critical for producing and recycling tetrahydrobiopterin. If one of the enzymes fails to function correctly because of a gene mutation, little or no tetrahydrobiopterin is produced. As a result, phenylalanine from the diet builds up in the bloodstream and other tissues and can damage nerve cells in the brain. High levels of phenylalanine can result in signs and symptoms ranging from temporary low muscle tone to mental retardation, movement disorders, difficulty swallowing, seizures, behavioral problems, progressive problems with development, and an inability to control body temperature.
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Absorption
No information avaliable
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side effects and Toxicity
No information avaliable
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Patient Information
No information avaliable
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Organisms Affected
Humans and other mammals