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.

Here is our schedule of webcasts. Check back often as we will continually update this information with names of guests and other details. Also, be sure to send in questions as you have them and sign up for our Special Edition newsletter and alerts. These programs will be available as replays for downloading, streaming and podcasting. Approximately 1 hour before the program begins, a link to the live stream will appear on this web page.

Ask a Question

Notice: Patient Power, LLC is solely responsible for the content of webcasts and replays and transcripts on this site. Information here is for educational purposes only. Please check with your own doctor for medical advice that is right for you.