Sponsored through an educational grant from BIOMARIN

New Approaches to Managing PKU

The rare genetic condition known as phenylketonuria (PKU) is characterized by an inability to metabolize the amino acid phenylalanine (Phe). Phe is toxic to the brain, and it can progressively produce serious neurological damage, mental retardation, reduced IQ and motor deficits if uncontrolled.

The Phe-Restricted Diet

For the past 40 years, PKU has been managed with a Phe - restricted diet that limits the intake of Phe to avoid the toxic effects on the brain. However, managing PKU with the phe-restricted diet is not as easy as it might sound. Patients have difficulty in adhering to the stringent diet, which excludes common foods with high protein content, such as meat, eggs, cheeses and beans. Lapses are common even among committed patients and Phe can find its ways into the blood stream even when the patient sincerely tries to maintain the diet. One study found that 59 percent of people who said they were strictly following the diet had higher than recommended Phe levels in their blood. In addition, the Phe-restricted diet can cause nutritional deficiencies that result in growth retardation and reduced bone mass.

Introducing: New Medicine for People with PKU

For the first time people with PKU have a medicine to help control their condition. KUVAN (sapropterin dihydrochloride) is the first prescription drug approved by the FDA to reduce blood Phe levels when used in conjunction with a Phe-restricted diet. The medication lowers phe levels. Lower phe levels lead to better medical outcomes and quality of life.

Learn more about the new approach to managing PKU in this important series of Patient Power programs featuring leading medical experts, dieticians and regular people successfully coping with the condition.

Listen to Coverage from the recent International Teen and Adult PKU conference held in Chicago in August 2008. This marked the largest ever assembled group of PKU patients and family members ever!

How to Listen

(Live from 8:00 pm to 9:00 pm Eastern on program dates)

Online: Approximately 1 hour before the program begins, a link to the live stream will appear on this web page.

Phone: Listen over the phone by dialing (877) 774-2465.

Questions: Email questions in advance or during the program to pku@patientpower.info.

Ask a Question

Living Better with PKU (Spring 2008 Series) Survey

We hope the information you received here has helped you to better manage PKU for you or a loved one.

To continue to develop programming that is responsive to your needs, we need input from you. Your responses to this Special Edition: Better Living with PKU Survey will tell us how well we've succeeded so far and how we can improve.

The survey is less than 20 questions and can be completed in just a few minutes. The information will be used only in aggregate and none of the responses will be indentified with an individual. We will ask only for your ZIP code and other basic information.

Thanks for taking the survey. Patient Power looks forward to developing future programs for the PKU community, and we hope you will be in the audience.