Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response
| Status: | Completed |
|---|---|
| Conditions: | Endocrine |
| Therapuetic Areas: | Endocrinology |
| Healthy: | No |
| Age Range: | 4 - Any |
| Updated: | 4/2/2016 |
| Start Date: | December 2008 |
| End Date: | February 2010 |
| Contact: | Louis J Elsas, MD |
| Email: | lelsas@med.miami.edu |
| Phone: | 305 243 7126 |
The investigators will investigate the effect of blood phenylalanine on Kuvan responsiveness
in the same patients with PKU when their blood phenylalanine concentrations are altered by
diet.
Lowering blood phenylalanine concentrations in Kuvan non-responsive patients with PKU will
increase the frequency of Kuvan responsiveness.
in the same patients with PKU when their blood phenylalanine concentrations are altered by
diet.
Lowering blood phenylalanine concentrations in Kuvan non-responsive patients with PKU will
increase the frequency of Kuvan responsiveness.
Inclusion Criteria:
- all patients with a diagnosis of phenylketonuria
- patients who are over the age of 4 years,
- patients with a fasting blood phenylalanine ≥600µmol/l
Exclusion Criteria:
- female patients who are pregnant or attempting to become pregnant
- children under four years of age
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