Into Science Project Project Highlights First And Only Drug To Treat Metabolic Condition, PKU (NAPSA)—Lauren Petrosh, age 13, decided to do her school science fair project on something Purpose Hypothesis very close to home—a genetic disorder she will live with for the rest of her life, and the therapy she’s taking to manageit. Lauren’s science fair project focused on a metabolic disorder called Phenylketonuria, or PKU, a condition that prevents the body from metabolizing an essential amino acid found in many common foods, including meat, eggs, whole grains and dairy. If these foods, containing the amino acid Phenylalanine, or Phe, are consumed, dangerously high levels of Phe build up in the bloodstream and are toxic to the brain. As a result, people with PKU, like Lauren, must follow a strict diet consisting of specially produced foods andartificial formulas to replace the nutrients lacking from the foods they cannoteat. Lauren recently began taking the first and only FDA-approved medicine for the treatment of PKU, called Kuvan™ (sapropterin dihydrochloride) Tablets, and she highlighted the results in her science fair project. Kuvan has been shown to lower blood Phelevels in some patients with PKU, thereby helping to lower the buildup of toxic levels of Phe in the blood. “I know that for people with PKU, keeping Phelevels low is very important. As my science fair project shows, taking Kuvan in addition to my Phe-restricted diet helped me reduce the Phe levels in my blood and achieve my goals for treatment,” said Lauren. People with PKU face severe brain-related problems if their diseaseis left untreated, including slower thinking, memory loss, concentration problems, mood dis- How Tuen Do —— = Lauren Petrosh used the FDAapproved medicine Kuvan in her project on a metabolic disorder called Phenylketonuria, or PKU. orders and, in some cases, severe mental retardation. Approximately 13,000 people in the U.S. live with this condition. Response to Kuvan will vary. Patients should work closely with their physicians and dietitians to evaluate whether the treatmentis right for them. “Tm glad there’s finally a medication for my PKU—it’s important for me to lower my levels of Phe, since dangerouslevels of Phe can cause several mental problems, and Kuvan helps me lower my Phelevels,” explained Lauren. “Through myscience fair project, I’m hoping to teach others about my condition and show that there is a treatment that works to lower blood Phe levels in some people like me.” To assist PKU patients with access to Kuvan, BioMarin Phar- maceutical, the maker of Kuvan, has established the BioMarin Patient and Physician Support Program, or BPPS. Through BPPS, every PKU patient eligible for Kuvan will be matched with a case manager who will help with access to the therapy. To learn more, visit www.kuvan.com orcall (877) MY KUVAN (1-877-6958826). w----------------------- eee PML ---------------------------Important Safety Information: Kuvan (sapropterin dihydrochloride) full prescribing information. BioMarin Pharmaceutical Inc. High blood Phelevels are toxic to the brain and can lead to lower intelligence and decrease in the ability to focus, rememberand organize information. Any change you maketo your diet may impact yourblood Phelevel. Follow your doctor’s instructions carefully. Your doctor and dietitian will continue to monitor and change your diet throughout your treatment with Kuvan. If you have a fever, or if you are sick, your Phe level may go up. Tell your doctor and dietitian as soon as possible so they can see if they have to adjust your treatment to help keep your blood Phe levels in the desired range. Kuvanis a prescription medicine and should not be taken by people whoareallergic to anyof its ingredients. Tell your doctor if you have ever had liver problems, are nursing or pregnant or may become pregnant, have poor nutrition or are anorexic. Your doctor will decide if Kuvanis right for you. Tell your doctor aboutall the medicines you take. The most common side effects reported when using Kuvan are headache, diarrhea, abdominal pain, upper respiratory tract infection (like a cold), throat pain, vomiting and nausea. To report SUSPECTED ADVERSE REACTIONS,contact BioMarin Pharmaceutical Inc. at (866) 906- 6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. Please visit www.kuvan.com for complete product information.
