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First-of-Its-Kind Study Suggests Importance of Routine Mental Health Screening in the Care of Patients Suffering From Genetic Disease PKU
Date:3/18/2011

apropterin Dihydrochloride (the active ingredient in KUVAN) on endpoints that include measures of global functioning and the symptoms of attention deficit hyperactivity disorder (ADHD), depression, anxiety and executive functioning in 200 individuals with PKU.  
  • PKU-015 Interim Report: A Phase IIIB Study of Sapropterin Dihydrochloride (KUVAN) in Young Children with PKU (N Longo, Pediatrics, University of Utah and A Feigenbaum, Hospital for Sick Children, Toronto, Ontario) [Poster 96 – March 19, 10:30 - 11:30 am].  The poster presentation included preliminary data from a Phase IIIb, open-label, clinical study evaluating the long-term efficacy and safety KUVAN in preserving neurocognitive function in young (age 0 to six years of age) children with PKU.  Data will include the first 80 patients, of the total expected approximately 100 patients, as the study is ongoing.

  • Copies of the scientific abstracts presented at the ACMG conference are available upon request by calling the BioMarin Corporate Communications Department.

    ADAPT Study Design and ResultsA total of 89 patients (42 adults and 47 children) over five years of age with a confirmed PKU diagnosis received screening for  psychiatric distress and cognitive impairment as part of the standard of care over a 10-month period. Results from this program demonstrate that 29 percent of patients screened positive for psychiatric distress (n=26 / 89), with more adults testing positive compared to children (43% vs.17%). In addition, those patients who screened positive had significantly higher mean blood Phe levels compared to those who screened negative (749+/-575 umol/L vs. 468+/-351 umol/L, P=.02).  Findings also showed that the older the patients, the higher the blood Phe levels (r2= .37; P<.001). 

    The study also demonstrated that patients who were being treated with diet plus KUVAN (n=36) had a lower mean blood Phe level c
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    SOURCE BioMarin Pharmaceutical Inc.
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