The following links are helpful online resources for your work with PKU patients.

  • BioMarin (BMRN.com)
    BioMarin develops and commercializes innovative biopharmaceuticals, including KUVAN, for serious diseases and medical conditions.
  • PKU.com
    PKU.com, sponsored by BioMarin, is a comprehensive online resource for the PKU community that supplies information about diet management, disease background, products in development, recipes, and health.
  • KDI Services
    KDI Services is a hotline for healthcare providers that offers access to expert dietitians who have experience managing patients taking KUVAN. KDI Services can answer your questions regarding nutritional management for your patients taking KUVAN. KDI Services is available to you Monday through Friday during normal business hours.

    The registered dietitians in KDI Services provide information to assist healthcare providers. This service is not meant to replace the care given by the providers. Healthcare providers are responsible for the patient's KUVAN treatment and all decisions related to patient care.

    You can contact KDI Services by phone through BioMarin's Medical Information Services at 1-800-983-4587 or by email at medinfo@BMRN.com. Please let us know the best way to respond to your inquire, by phone or email, and the preferred number, date and time to call back. A response to your inquiry will be made within 24 hours.
  • BH4.org
    BH4.org provides scientific literature downloads, conference information, database links, and current news articles related to PKU.
  • PAH Database (pahdb.mcgill.ca)
    Provides users with access to up-to-date information about mutations at the PAH locus, including access to the content of the database via electronic reports.
  • PKU Organization of Illinois (pkuil.org)
    Publishes a newsletter on a quarterly basis that serves as a forum for the exchange of new information, ideas, recipes, and encouragement. Sponsors PKU cooking schools.
  • The Children's PKU Network (www.PKUnetwork.org)
    The Children's PKU Network (CPN) is a not-for-profit organization dedicated to providing support and services to children with PKU, their families, and all those involved in the treatment of this disorder. Provides a variety of educational and support materials.
  • National PKU News Organization (PKUnews.org)
    National PKU News is a not-for-profit organization devoted to providing news and information to all persons involved in the treatment of PKU. Publishes educational materials regarding treatment of PKU. Food lists and cookbooks are also distributed by the organization.
  • Mid-Atlantic Connection for PKU and Allied Disorders (macpad.org)
    The Mid-Atlantic Connection for PKU and Allied Disorders (MACPAD) provides a network of support for families of newly diagnosed children and for individuals of all ages. It encourages proper treatment by providing nutritional guidance and information.
  • 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.
  • The Genetic Alliance (geneticalliance.org)
    The Genetic Alliance in Washington, D.C. is a coalition of more than 600 advocacy organizations serving 25 million people affected by 1000 conditions.
  • The National Society for phenylketonuria (nspku.org)
    The National Society for phenylketonuria (NSPKU) actively promotes the care and treatment of PKU and works closely with medical professionals in the UK. It organizes conferences, events, and research throughout the UK, posts a wide range of publications (including food lists) for parents, PKU patients, and medical professionals.
  • Taste Connections (www.tasteconnections.com)
    Taste Connections specializes in low-protein products such as bread mix, multi-baking mix, etc, that are suitable for patients with PKU or homocystinuria (HCU).
  • Lowprotein.com
    A Web site specializing in low-protein recipes for persons with HCU, PKU, methylmalonicacidemia (MMA), and tyrosinemia.
  • 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.
  • 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 how screening with mass spectrometry is performed, explains the genetics behind the results, and gives specific background information on PKU.
  • 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.
  • 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 in 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 indicated to reduce blood phenylalanine (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

Prolonged exposure to elevated blood Phe levels in PKU patients can result in severe neurologic damage. The initiation of KUVAN therapy does not eliminate the need for careful monitoring of blood Phe levels and ongoing dietary management.

Some patients receiving KUVAN can experience significant drops in blood Phe levels. Patients should be monitored closely to ensure that blood Phe levels do not fall too low.

Not all patients with PKU respond to treatment with KUVAN. Response to treatment can only be determined by a therapeutic trial of KUVAN.

KUVAN has not been studied in patients with liver or renal impairment. Patients who have these conditions should be carefully monitored when receiving KUVAN. Caution should be used with the administration of KUVAN to patients who are receiving levodopa and drugs that affect nitric oxide–mediated vasorelaxation or folate metabolism.

The most serious adverse reactions reported during KUVAN administration (regardless of relationship to treatment) were gastritis, spinal cord injury, streptococcal infection, testicular carcinoma, and urinary tract infection. Mild to moderate neutropenia was also noted. The most common adverse reactions were headache, diarrhea, abdominal pain, upper respiratory tract infection, pharyngolaryngeal pain, vomiting, and nausea.

Please read the full Prescribing Information by clicking here.

BIOMARIN