Support

The following online resources provide lots of important information about PKU and how it’s managed.

  • PKU.com
    Sponsored by BioMarin, PKU.com is your PKU connection to basic information, tips, and aids to help with PKU management, and to the diverse online PKU community. Create a free account with exclusive features and content, such as discussion forums, customized recipes, a personalized message inbox, and more.
  • The National Organization for Rare Disorders (rarediseases.org)
    The National Organization for Rare Disorders (NORD), Inc. is a federation of health organizations, patients, healthcare providers, and individuals. NORD is dedicated to helping people with rare “orphan” diseases and to assisting the organizations that serve them. NORD is committed to improving the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.
  • 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.
  • Screening, Technology And Research in Genetics (STAR-G) Project (newbornscreening.info/index)
    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 about genetics, how screening with mass spectrometry is performed, and specific background information on PKU.
  • The Mid-Atlantic Connection for PKU and Allied Disorders (Macpad.org)
    A non-profit organization that promotes better health and well-being in individuals with PKU and related metabolic disorders by providing information and facilitating the exchange of ideas within the PKU community.
  • 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.
  • Unified to Support Metabolic Disorders (USMD-PKU), Inc. (usmd-pku.org)
    The purpose of this non-profit organization is to bring together individuals, metabolic support groups, and professionals across America who are directly involved with issues related to inborn errors of metabolism requiring a low-protein diet, including PKU and other related metabolic disorders, to improve identification, treatment, and management of these disorders.
  • Mayo Clinic (MayoClinic.com)
    The Mayo Clinic is a not-for-profit medical practice devoted to the treatment of complex illnesses. The Web site, MayoClinic.com, provides an overview of PKU, including topics such as treatment, prevention, self-care, and coping skills.
  • PKU Adults and Teens (E-mail: pkuadult@aol.com)
    Start a conversation by sending a single e-mail. Write what’s on your mind, send it off, and let the discussion begin!
  • PKU Adults (pkuadults.com)
    This site provides links to resources and information on PKU, PKU symptoms, and DNA tests.
  • PKU World Link (pkuworldlink.org)
    PKU knows no boundaries. Find out where people with PKU live; you can also find a pen-pal with PKU in your state or in another country. A thorough history of the discovery of PKU is also presented.
  • State of Michigan (Michigan.gov)
    The state of Michigan’s Web site provides PKU screening information for newborns.
  • PKU Organization of Illinois (PKUIL.org)
    The PKU Organization of Illinois Web site features a broad range of PKU information, including clinics and product suppliers.
  • 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.
  • University of Washington PKU Clinic (depts.washington.edu)
    The University of Washington’s PKU Clinic Web site provides in-depth information on PKU and clinic insights from the Seattle PKU clinic.
  • US Food and Drug Administration Food and Nutrition Service (fns.usda.gov)
    Click on the title "Guidance" to find "Accommodating Children with Special Dietary Needs in the School Nutritional Programs." Click on this and you will be able to read about the provisions for PKU in the US as required by law.
  • Taste Connections (tasteconnections.com)
    Taste Connections specializes in low-protein products, such as bread mix and multibaking mix, that are suitable for patients with PKU or homocystinuria (HCU). The recipes were created by the mother of a patient with HCU who has a master’s degree in Nutritional Science and who presents workshops on low-protein cooking.
  • Low-Protein (lowprotein.com/about.htm)
    Started by the same woman who started Taste Connections, this site provides more low-protein food recipes for persons with HCU, PKU, methylmalonic acidemia (MMA), and tyrosinemia.
  • The California Late Treatment Project (pkulatetx.com/index.htm)
    This site provides treatment options for patients with PKU, regardless of age, with the goal of increasing the quality of life for these individuals and stopping the neurological deterioration that occurs in those not treated. The downloadable Late Treatment Protocol also has a handy list of Phe amounts found in over-the-counter and prescription medications.
  • Canadian PKU and Allied Disorders (canpku.org)
    Based in Toronto, Ontario, this non-profit organization is dedicated to providing up-to-date, accurate news and information to families and professionals dealing with phenylketonuria.

 

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 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.

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