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The Pursuit of PKU

This short film details the history of phenylketonuria (PKU) and BioMarin’s 15+ year commitment to advancing the standard of care for those living with this rare condition. Watch "The Pursuit of PKU” to learn more about BioMarin’s dedication to pioneering the understanding and treatment of PKU from team leaders and members of the community. -- BioMarin is a global biotechnology company focused on developing first-in-class therapeutics that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases. http://www.BioMarin.com   / biomarinofficial     / biomarin     / biomarinofficial   PALYNZIQ® (Pal-lin-zeek) (pegvaliase-pqpz) is a prescription medication used to lower blood levels of phenylalanine (Phe) in adults with PKU (phenylketonuria) who have uncontrolled blood Phe levels above 600 micromol/L (10 mg/dL) on their current treatment. You should discuss the potential benefits and risks of PALYNZIQ with your healthcare provider. What is the most important information I should know about PALYNZIQ? PALYNZIQ can cause a severe allergic reaction (anaphylaxis) that may be life threatening and can happen any time during treatment with PALYNZIQ. Severe allergic reactions are a serious but common side effect of PALYNZIQ. • You will receive your first injection of PALYNZIQ in a healthcare setting where you will be closely watched for at least 1 hour after your injection for a severe allergic reaction • Your healthcare provider will prescribe auto-injectable epinephrine for you, and will teach you (or your caregiver) and your observer, if needed, when and how to use it if you have a severe allergic reaction • If you have a severe allergic reaction during treatment with PALYNZIQ, you will need to receive an injection of epinephrine immediately and get emergency medical help right away • Your healthcare provider will decide if you (or your caregiver) are able to give the PALYNZIQ injections, recognize the signs and symptoms of a severe allergic reaction, give an injection of epinephrine, and call for emergency help, if needed • Your healthcare provider may recommend that an adult observer (or your caregiver) be with you when you give your PALYNZIQ injection and for at least 1 hour after your injection to watch you for signs and symptoms of a severe allergic reaction and, if needed, give you an injection of epinephrine and call for emergency medical help The most common side effects of PALYNZIQ include injection site reactions (such as redness, itching, pain, bruising, rash, swelling, or tenderness), joint pain, headache, skin reactions that spread and last at least 14 days (such as itching, rash, or redness), nausea, stomach pain, vomiting, cough, mouth and throat pain, itching, diarrhea, stuffy nose, feeling very tired, dizziness, anxiety, and low levels of Phe in your blood. These are not all of the possible side effects of PALYNZIQ. Speak with your healthcare provider right away about any side effects. You may report side effects to BioMarin at 1-866-906-6100. Please see additional important safety information here: https://www.palynziq.com/#isi-container You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. Please see full Prescribing Information at http://bit.ly/Palynziq-Prescribing-Info, including an important warning for risk of anaphylaxis, and the Medication Guide at http://bit.ly/Palynziq-Med-Guide.

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