Support

Below are some helpful links for parents who want to know more about their child’s PKU diagnosis. A more complete list of links can be found by clicking here.

• PKU.com
  Sponsored by BioMarin, PKU.com is your connection to basic information, tips, and aids to help with PKU management, and to the diverse online PKU community. Create a free account for exclusive features and content such as discussion forums, customized recipes, a personalized message in-box, and more.
• Children’s PKU Network (PKUnetwork.org)
  This non-profit organization is devoted to addressing the unique needs of children with PKU and their families by promoting awareness and by providing education and assistance programs.
• State of Michigan (Michigan.gov)
  The state of Michigan’s Web site provides information about PKU screening of newborns.
• National PKU News Organization (PKUnews.org)
  This non-profit organization provides the latest PKU news and information to people affected by PKU and their families.
• Texas Department of State Health Services (dshs.state.tx.us/newborn/pku.shtm)
  The Newborn Screening Case Management Program of Texas provides customer-oriented, high-quality newborn screening follow-up, case management, and outreach education. It has a section about PKU for parents and provides a Spanish translation as well.
• Screening, Technology and Research in Genetics (STAR-G) Project (newbornscreening.info/index.html)
  The goal of the STAR-G project is to examine the financial, ethical, legal, and social issues posed by the use of tandem mass spectrometry in newborn screening. It explains how screening with mass spectrometry is performed, the genetics behind the results, and gives specific background information on PKU.

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Indication

KUVAN^® (sapropterin dihydrochloride) Tablets is approved to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is to be used in conjunction with a Phe-restricted diet.

Important Safety Information

High blood Phe levels are toxic to the brain and can lead to lower intelligence and decrease in the ability to focus, remember and organize information. Any change you make to your diet may impact your blood Phe level. Follow your doctor’s instructions carefully. Your doctor and dietitian will continue to monitor and may adjust your diet throughout your treatment with KUVAN.

If you have a fever, or if you are sick, your Phe level may go up. Tell your doctor and dietitian as soon as possible so they can see if they have to adjust your treatment to help keep your blood Phe levels in the desired range.

KUVAN is a prescription medicine and should not be taken by people who are allergic to any of its ingredients. Tell your doctor if you have ever had liver or kidney problems, are nursing or pregnant or may become pregnant, have poor nutrition or are anorexic. Your doctor will decide if KUVAN is right for you. Tell your doctor about all the medicines you take.

The most common side effects reported when using KUVAN are headache, diarrhea, abdominal pain, upper respiratory tract infection (like a cold), throat pain, vomiting, and nausea.

To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please read the full Patient Information by clicking here.