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Patient Stories
Lauren has PKU [5:35 minutes]
 Lauren is a very active teenage: she sings in the church choir, plays on the basketball and tennis teams at school, is secretary of her student council, and is in the National Junior Honor Society. Lauren recently won first place at her school's science fair. Her project was about charting her blood Phe levels before and after KUVAN.
When her parents found out that Lauren had PKU 19 days after birth, they were referred to the PKU clinic at Tulane. There they learned the importance of sticking to diet, and as a small child, Lauren had blood tests every week to ensure her blood Phe levels were within normal, healthy range. Last fall, Lauren started on KUVAN, and during the first week of treatment, her blood Phe levels dropped substantially.
Greg has PKU [4:30 minutes]
 Greg loves to lift weights, read, and practice martial arts. It wasn't always that way. When Greg was born, he had elevated Phe, but it was not high enough to be considered classical PKU. Greg was on a special low-Phe formula until the age of 3.
However, the warning signs were there. He had trouble concentrating and struggled academically in school. When he started working out and eating a high protein diet in his late teens, Greg began to feel irritable and frustrated, and started getting headaches. At about 26, he began to experience serious anxiety and depression.
It was at that point that his father told him the doctors thought he had PKU as a baby. Sure enough, a filter paper test came back positive for PKU. Greg is on a low-Phe diet and KUVAN, and now and his Phe levels are lowered and under control.
Shayna has PKU [4:50 minutes]
 Shayna is a fun-loving girl who likes birthday parties. She is a big sister and is just starting school. Her mother found out that Shayna had PKU a week after she was born, when the newborn screening test came back positive. Shayna immediately went on a low-Phe diet and her mother started educating herself about PKU and found their doctor, Dr. Burton.
There have been struggles like the phase when Shayna was three and started refusing to take her food medication. But the family got past that. Since then Shayna has been able to keep Phe levels consistent and within range with the help of diet and KUVAN. Shayna and her mother talk about their experiences with PKU, which center around food: birthdays with sherbet cake, family meals and shopping, and favorite foods dining out.
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Indication
KUVAN
® (sapropterin dihydrochloride) Tablets are approved to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is to be used in conjunction with a low-Phe diet.
Important Safety Information
High blood Phe levels are toxic to the brain and can lead to lower intelligence and decrease in the ability to focus, remember and organize information. Any change you make to your diet may impact your blood Phe level. Follow your doctor's instructions carefully. Your doctor and dietitian will continue to monitor your diet and blood Phe levels 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.
KUVAN is a prescription medicine and should not be taken by people who are allergic to any of its ingredients. Tell your doctor if you have ever had liver or kidney problems, are nursing or pregnant or may become pregnant, have poor nutrition or are anorexic. Your doctor will decide if KUVAN is right for you. Tell your doctor about all 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 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please read the full Patient Information by clicking here.
