Pediatric Genetics - Research

We are currently involved in several research studies for patients with PKU (Phenylketonuria). All of these studies have something to do with Kuvan and are sponsored by BioMarin. One is a registry, which collects information about how patients are doing. This registry is for patients who have tried, are taking, or are planning to try Kuvan soon.

The next study is for patients 0-6 years of age, and is looking at information about safety, blood Phe levels, neurocognitive function (how the brain is working), and growth for patients with PKU who are responders to Kuvan.

The third study has just opened and is for patients 12 years of age and older. This study is also to gather information about the safety of taking Kuvan and the effect on Phe levels. In addition, information about the neurocognitive effects (how the brain is working), and effects on signs of ADHD, depression, and anxiety will be studied.

If you are interested in finding out more about the studies, please contact Heidi Watts at 531-0003 extension 283765, and tell her you want to discuss research studies with Kuvan.

Penn State Hershey Pediatric Clinical Research Office (PCRO) is to enhance, foster and promote organized clinical research within the Penn State Children's Hospital. By stimulating clinical research, the Pediatric Clinical Research Office will help Penn State Milton S. Hershey Medical Center meet its goals of patient care, teaching, service and research. The PCRO is currently and strives to continue to be active in PSU investigator-initiated research through collaborations between faculty and our office and its research staff. The PCRO helps investigators initiate new studies and provides recruitment, consent writing, regulatory, budgetary, and data collection assistance where needed. This help may also include administrative, procedural, and case report documentation. Help is also provided to investigators with industry-sponsored clinical trials. The PCRO promotes investigator initiated research within central Pennsylvania, trains our staff to promote quality research within the Hershey Medical Center and the adoption of “Good Clinical Practices."  More...