SAPROPTERIN DIHYDROCHLORIDE
PHENYLKETONURIA TX - COFACTORS OF PHENYLALANINE HYDROXYLASE·PHENYLKETONURIA
AUROBINDO PHARM
Indication
Phenylketonuria
Phenylketonuria is an autosomal recessive inborn error of metabolism characterized by a deficiency in the hepatic enzyme phenylalanine hydroxylase, which converts phenylalanine into tyrosine. Read more about Phenylketonuria
13 drugs 9 manufacturers
Drugs that treat it
Common Phenylketonuria therapies.
SAPROPTERIN DIHYDROCHLORIDE
PHENYLKETONURIA TX - COFACTORS OF PHENYLALANINE HYDROXYLASE·PHENYLKETONURIA
Hikma Pharmaceuticals USA
SAPROPTERIN DIHYDROCHLORIDE
PHENYLKETONURIA TX - COFACTORS OF PHENYLALANINE HYDROXYLASE·PHENYLKETONURIA
TRUPHARMA, LLC.
SAPROPTERIN DIHYDROCHLORIDE
PHENYLKETONURIA TX - COFACTORS OF PHENYLALANINE HYDROXYLASE·PHENYLKETONURIA
MICRO LABS USA,
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Coming soonPhenylketonuria is an autosomal recessive inborn error of metabolism characterized by a deficiency in the hepatic enzyme phenylalanine hydroxylase, which converts phenylalanine into tyrosine.
This defect leads to the systemic accumulation of phenylalanine and its metabolites in the blood and tissues, primarily impacting the central nervous system.
Typically identified during newborn screening, untreated disease manifests clinically with profound and irreversible intellectual disability, microcephaly, developmental delay, seizures, behavioral abnormalities, hypopigmentation, and a characteristic musty body odor.
- nih.gov
Phenylketonuria is an autosomal recessive inborn error of metabolism characterized by a deficiency in the hepatic enzyme phenylalanine hydroxylase, which converts phenylalanine into tyrosine This defect leads to the systemic accumulation of phenylalanine and its metabolites in the blood and tissues, primarily impacting the central nervous system
- nih.gov
Phenylketonuria is an autosomal recessive inborn error of metabolism characterized by a deficiency in the hepatic enzyme phenylalanine hydroxylase, which converts phenylalanine into tyrosine
- nih.gov
This defect leads to the systemic accumulation of phenylalanine and its metabolites in the blood and tissues, primarily impacting the central nervous system
- medlineplus.gov
Typically identified during newborn screening, untreated disease manifests clinically with profound and irreversible intellectual disability, microcephaly, developmental delay, seizures, behavioral abnormalities, hypopigmentation, and a characteristic musty body odor
- medlineplus.gov
Typically identified during newborn screening, untreated disease manifests clinically with profound and irreversible intellectual disability, microcephaly, developmental delay, seizures, behavioral abnormalities, hypopigmentation, and a characteristic musty body odor
- medlineplus.gov
Typically identified during newborn screening, untreated disease manifests clinically with profound and irreversible intellectual disability, microcephaly, developmental delay, seizures, behavioral abnormalities, hypopigmentation, and a characteristic musty body odor
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