Are you a healthcare professional?

Yes   No


Your name:
Your email:
Friend's emails:
(multiple emails separated by comma)
Your message:
 
Information for Patients & Caregivers
|

References

Search

Share

References

  1. Mitchell J, Scriver CR. Phenylalanine hydroxylase deficiency. Bethesda (MD): National Library of Medicine (US), National Center for Biotechnology Information; Gene Reviews. 2007 March. Available from: http://www.genetests.org/query?dz=pku. Accessed February 3, 2009.
  2. Scriver CR, Kaufman S. Hyperphenylalaninemia: phenylalanine hydroxylase deficiency. In: Scriver CR, Beaudet AR, Sly W, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease. 8th ed. New York, NY: McGraw-Hill; 2001:1667-1724.
  3. Waisbren SE, Noel K, Fahrbach K, et al. Phenylalanine blood levels and clinical outcomes in phenylketonuria: a systematic literature review and meta-analysis. Mol Genet Metab. 2007;92:63–70.
  4. Moyle JJ, Fox AM, Arthur M, Bynevelt M, Burnett JR. Meta-analysis of neuropsychological symptoms of adolescent and adults with PKU. Neuropsychol Rev. 2007;17:91-101.
  5. VanZutphen KH, Packman W, Sporri L, et al. Executive functioning in children and adolescents with phenylketonuria. Clin Genet. 2007;72:13-18.
  6. Pietz J, Fatkenheuer B, Burgard P, Armbruster M, Esser G, Schmidt H. Psychiatric disorders in adult patients with early-treated phenylketonuria. Pediatrics. 1997;99(3):345-350.
  7. Kaufman, S. A model of human phenylalanine metabolism in normal subjects and in phenylketonuric patients. Proc. Natl. Acad. Sci. 1999;96:3160-3164.
  8. Anastasoaie V, Kurzius L, Forbes P, and Waisbren S. Stability of blood phenylalanine levels and IQ in children with phenylketonuria. Mol Genet Metab. 2008;95(1-2):17-20.
  9. National Institutes of Health Consensus Development Panel.National Institutes of Health Consensus Development Conference Statement: phenylketonurioa: screening and management, October 16-18, 2000. Pediatrics. 2001:108:972-982.
  10. Waisbren SE, Noel K, Fahrbach K, et al. Phenylalanine blood levels and clinical outcomes in phenylketonuria: a systematic overview and meta-analysis. Mol Genet Metab. 2007;92:63-70.
  11. Przyrembel H, Bremer HJ. Nutrition, physical growth, and bone density in treated phenylketonuria. Eur J Pediatr. 2000;159(suppl 2):S129-S135.
  12. KUVAN prescribing information, BioMarin Pharmaceutical Inc.
  13. Trefz FK, Burton BK, Longo N, Casanova MM, Gruskin DJ, Dorenbaum A, Kakkis ED, Crombez EA, Grange DK, Harmatz P, Lipson MH, Milanowski A, Randolph LM, Vockley J, Whitley CB, Wolff JA, Bebchuk J, Christ-Schmidt H, Hennermann JB; Sapropterin Study Group. Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study.
    J Pediat. 2009 May;154(5):700-7.
  14. Steinfeld R, Kohlschütter A, Ullrich K, Lukacs Z. Efficiency of long-term tetrahydrobiopterin monotherapy in phenylketonuria. J Inherit Metab Dis. 2004;27:449-453.
  15. Shintaku H, Kure S, Ohura T, et al. Long-term treatment and diagnosis of tetrahydrobiopterin responsive hyperphenylalaninemia with a mutant phenylalanine hydroxylase gene. Pediatr Res. 2004;55:425-430.
  16. Hennermann JB, Bührer C, Blau N, Vetter B, Mönch E. Long-term treatment with tetrahydrobiopterin increases phenylalanine tolerance in children with severe phenotype of phenylketonuria. Mol Genet Metab.
    2005;86(suppl 1):S86-S90.
  17. Trefz FK, Scheible D, Frauendienst-Egger G, Korall H, Blau N. Long-term treatment of patients with mild and classical phenylketonuria by tetrahydrobiopterin. Mol Genet Metab. 2005;86(suppl 1):S75- S80.
  18. Koch R, Azen C, Friedman EF, Williamson MS. Paired comparisons between early treated PKU children and their matched sibling controls on intelligence and school achievement test results at eight years of age. J. Inherit Metab Dis. 1984l7L86-90.
  19. Walter JH, White FJ, Hall SK et al. How practical are recommendations for dietary control in phenylketonuria? Lancet. 2002;360:55-57.
  20. Waisbren SE, Hanley W, Levy HL, et al. Outcome at age 4 years in offspring of women with maternal phenylketonuria: the Maternal PKU Collaborative Study. JAMA. 2000;283(6):756-762.

Indication

KUVAN® (sapropterin dihydrochloride) Tablets are 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 low-Phe 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 your diet and blood Phe levels 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.


This site is intended for use by U.S. residents only.

KUVAN® is a registered trademark of BioMarin Pharmaceutical Inc. Developed in collaboration with Asubio Pharma Co., Ltd.
©2010 BioMarin Pharmaceutical Inc. All rights reserved.