12
Participants
Start Date
August 31, 2011
Primary Completion Date
August 31, 2013
Study Completion Date
August 31, 2013
Sapropterin
Sapropterin will be taken daily for 12 or 24 weeks. Starting dose will be 20mg/kg/day and will increase at the 8 week visit to 30 mg/kg/day. Dosing may be further increased to as high as 40 mg/kg/day in attempt to normalize BH4 levels in CSF. Starting dose for Cohort 2 will be determined from data analysis in Cohort 1.
University of Utah, Salt Lake City
Collaborators (1)
BioMarin Pharmaceutical
INDUSTRY
University of Utah
OTHER