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PKU Research

There are many exciting things happening in the world of PKU research! Below is a sampling of some current and recent research, as well as links for more information.

National PKU Alliance

The National PKU Alliance (NPKUA) works to improve the lives of individuals and families associated with phenylketonuria (PKU) through research, support, education, and advocacy, while ultimately seeking a cure.

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Kuvan™ (sapropterin dihydrohchloride), formerly known as Phenoptin™, is an investigational small-molecule oral therapeutic for the treatment of PKU. Sapropterin dihydrochloride, the active ingredient in Kuvan, is a synthetic form of 6R-BH4. 6R-BH4 is an essential enzyme cofactor that works in conjunction with phenylalanine hydroxylase to metabolize Phe. For more information on the Phase III clinical trials of Kuvan go to

Scripps Research Institute

The Stevens Laboratory at Scripps Research Institute is focusing on creating a once or twice per week injectable enzyme therapeutic for classical PKU advanced. It is now in preclinical studies at BioMarin Pharmaceuticals. They have obtained U.S. National Institutes of Health (NIH) funding to convert and improve the injectable therapeutic into an orally available therapeutic.

Researchers at the Stevens Laboratory believe the potential conversion of an injectable therapeutic to an orally available therapeutic will be critical for those individuals with classical PKU, allowing them to improve their quality of life, as regular injections over a sustained period of time can be difficult for almost any patient. It is possible that the methods used to create the orally available therapeutic can also be applied to other metabolic disorders currently treated with injectable therapeutics. For more information visit

Kansas State University

Research in the Department of Plant Pathology at Kansas State University is focusing on producing a genetically engineered soybean that is free of phenylalanine, as a source of dietary protein for those who with PKU and related disorders. This project is partially funded through MACPAD and the PKU Organization of Illinois. For more information visit

Clinical Trials in Progress

To read what current clinical trials are in progress visit the FDA site by clicking here.