Human Genetics Growth and Development

PKUDOS - PKU Demographics, Outcomes and Safety Registry (IRB# 29641)

Status: Active

Investigators:

  • Patricia Gordon, MD

Categories:

  • Human Genetics Growth and Development
 

(PKU015) A Phase 3b Open-Label Study to Evaluate the Effect of Kuvan on Neurocognitive Function, Maintenance of Blood Phenylalanine Concentrations, Safety, and Population Pharmacokinetics in Young children with Phenylketonuria. (IRB# 30839)

Status: Active

Investigators:

  • Patricia Gordon, MD

Categories:

  • Human Genetics Growth and Development
 

(PKU016) A double-blind, placebo-controlled, randomized study to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with phenylketonuria. (IRB# 34121)

Status: Active

Investigators:

  • Patricia L. Gordon, MD

Categories:

  • Human Genetics Growth and Development