Innovative therapeutics



Kuvan® (sapropterin dihydrochloride) Patient
Yorktown, New York

Liam - BioMarin Patient StoriesLiam is happiest when he’s playing soccer, Legos, or with his dog Finn. He says he’s got huge plans for the future. “I want to be an oceanographer like Robert Ballard and raise the Titanic” he says. “I’ll make a movie and probably hire Harrison Ford for the leading role.”

His mother explains the difficulties the family had before they were able to bring Liam’s Phe levels under control.

“Life revolved around him, his formula, and his food,” says his mother Rosie. “There was no balance in our home because we were focused on trying to control his Phe. He was unhappy and struggling, so we were unhappy and struggling.”

Dynamics at home are much different today, however.

Liam and family - BioMarin Patient Stories“We continue to weigh all of Liam’s food and he still maintains a low protein diet, but now with Kuvan he feels like he has added help in his battle to manage his Phe,” Rosie says.

Liam has been on Kuvan therapy since December 2007. “My life is pretty good now that my Phe levels are lower,” he says.

Before starting therapy, Liam’s blood Phe levels peaked at 7 mg/dL, but today, by both maintaining a low-Phe diet and taking his daily dose of Kuvan, he’s holding steady at about 4 mg/dL.*

“Before Kuvan, I understood my diet, but I didn’t really cooperate because I didn’t like food being taken away. I would feel angry and sad because I couldn’t have any foods I like,” he recalls. “I’m still eating the same food as before, but since I started taking Kuvan, I’ve been more cooperative because I feel good that my Phe is lower,” he continues; “basically I am a success story.”

“I think Liam is happier now,” recalls his sister, Catriona. “He is eating better and does more activities outside.”

Now, with Phe levels under control, Rosie says that Liam has been better able to deal with his low-protein PKU diet.

“In the days before Kuvan, Liam had a can-do attitude in all areas of life except his diet. Now that attitude is applied to dietary compliance and managing his Phe levels,” she says. “He’s more compliant with his diet and he’s taking his vitamin supplements and medical formula. Now he’s easily getting about 60 grams of protein each day from formula, while in the past, every ounce was a struggle.”

He still eats the same low-protein PKU foods, like rice and spaghetti, but now he can also eat some foods with more natural protein, Rosie says. Occasionally, Liam can even enjoy a small portion of his favorite food, fava beans.

“I am delighted that Liam’s Phe levels are better now,” Rosie says, “and most days are good—days when I know Liam has had enough Phe, protein, and calories and we can all go to bed satisfied.”

* Individual patient response to Kuvan® therapy will vary.

Full Indication and Important Safety Information
Kuvan® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution is approved to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used with a Phe-restricted 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

Kuvan is available by prescription only. To learn more, please visit for full prescribing information.  If you have any questions about this information, please talk with your doctor.

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Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

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