What is sapropterin used for?
Sapropterin is used to lower phenylalanine levels in the blood of patients with phenylketonuria (PKU). High levels of phenylalanine (an amino acid) in the blood can cause severe brain damage, including severe mental retardation, seizures, tremors, or decreased learning ability.
Is sapropterin a drug?
Sapropterin is in a class of medications called cofactors. It works by helping the body to break down phenylalanine so it will not build up in the blood.
Can people with PKU eat tyrosine?
Persons with phenylketonuria (PKU) cannot synthesize tyrosine from phenylalanine because of a severe deficiency of the hepatic enzyme phenylalanine hydroxylase (phenylalanine 4-monooxygenase). Therefore, in these persons, tyrosine is an essential amino acid.
What does kuvan do for PKU?
KUVAN adds more BH4 and stimulates the PAH enzyme to process Phe in people with PKU. The PAH enzyme begins to convert Phe to tyrosine, and helps to keep your blood Phe levels low.
What is the life expectancy of a person with phenylketonuria?
PKU does not shorten life expectancy, with or without treatment. Newborn screening for PKU is required in all 50 states. PKU is usually identified by newborn screening. A child’s outlook is very good if she strictly follows the diet.
What is PKU diet?
The diet for PKU consists of a phenylalanine-free medical formula and carefully measured amounts of fruits, vegetables, bread, pasta, and cereals. Many people who follow a low phenylalanine (phe) food pattern eat special low protein breads and pastas.
What is Kuvan used to treat?
This medication is used along with diet changes for long-term treatment of a certain inherited disorder (phenylketonuria-PKU). It helps remove a certain chemical (phenylalanine) from the body. Too much phenylalanine in the body can cause brain damage.
What is the drug sapropterin used for in PKU?
Phenylketonuria (PKU) is a disorder in which the body cannot break down phenylalanine. Elevated phenylalanine levels can cause problems with thinking and behavior. Sapropterin is used to lower blood levels of phenylalanine in people with a certain type of PKU.
What is the difference between sapropterin and phenylketonuria?
Sapropterin is a synthetic form of a natural substance that activates an enzyme needed to break down phenylalanine in the body. Phenylketonuria (PKU) is a disorder in which the body cannot break down phenylalanine. Elevated phenylalanine levels can cause problems with thinking and behavior.
What is the drug sapropterin used to treat?
Sapropterin is used along with a restricted diet to control blood phenylalanine levels in people who have phenylketonuria (PKU; an inborn condition in which phenylalanine may build up in the blood and causes decreased intelligence and a decreased ability to focus, remember, and organize information).
How is phenylketonuria (PKU) treated?
The first treatment for PKU. Kuvan ® (sapropterin dihydrochloride) Tablets and Powder for Oral Solution is the first FDA-approved medication for phenylketonuria (PKU). Kuvan is a form of BH4, the cofactor of the PAH enzyme, which helps the enzyme break down Phe. Kuvan is to be used in conjunction with a Phe-restricted diet.