Does your child have PKU (Phenylketonuria)? Let's talk about Sapropterin powder!

Sometimes, our children and we ourselves have to live with some special health conditions, right? One such condition is called Phenylketonuria (also known as PKU for short). One of the medicines given for this condition is Sapropterin. So today, let's learn about this medicine called Sapropterin, just like talking to a friend.

What is this medicine? (What is Sapropterin?)

Simply put, Sapropterin is a medication that helps control the excessive accumulation of a chemical called phenylalanine in the body of people with a condition called Phenylketonuria, or PKU. Remember, the small pieces of protein in the food we eat are called amino acids? Phenylalanine is one such amino acid. A person with PKU cannot properly break down this phenylalanine, that is, digest it and use it as needed. Then it accumulates in the blood and can damage the brain and nervous system over time.

This Sapropterin medicine helps break down that phenylalanine in the body. Then, its accumulation in the blood decreases. Often, along with this medicine, doctors advise a special diet and sometimes exercise. Because, in this situation, you need to reduce the amount of food containing phenylalanine as much as possible.

Doctors may also prescribe this medicine for other conditions, so if you have any questions, it's best to ask your doctor or pharmacist .

You can usually buy this medicine from pharmacies under the brand names JAVYGTOR or KUVAN.

Things to tell your doctor before taking this medicine

Before you start taking Sapropterin, you should definitely tell your doctor about these things. Because only then can they determine whether this medicine is right for you and how much to give you. Hiding this information will only cause you problems.

• Do you have any eating disorders ? That is, conditions such as anorexia (a mental state of losing weight by not eating) and bulimia (a condition of binge eating and then vomiting).
• Do you have a fever ?
• Do you have any infection ?
• Do you have kidney disease ?
• Liver diseaseis there?
• Do you have a condition that makes you feel like you're having a fit ( seizures ) ? Or have you had one before?
• Have you ever had an allergy to Sapropterin, another medication, food, food dyes, or preservatives?
• If you are pregnant or planning to become pregnant (this is very important).
• If you are a breastfeeding mother .

It's very important for your own health to tell the doctor these things without hiding them, right?

How do you use this medicine?

Okay, now let's see how to use this medicine exactly. This is an oral medication. It should be taken with a meal. You should follow the instructions on the prescription label that your doctor has given you. Never take more or less than the amount your doctor has told you.

This comes as a powder. You should drink it by dissolving it in water or apple juice. Ask your doctor or pharmacist for the exact amount of water or apple juice you should take. The powder should dissolve quickly, even completely. You should drink the medicine within 30 minutes of dissolving it. Do not leave it for longer than that.

Another thing, you can also mix a small amount of this powder into applesauce (like applesauce) or pudding. Ask your doctor about that too, if it's comfortable for you to do so.

Take your medicine on time, at the same time every day if possible. Don't take more than your doctor tells you to. Also, don't stop taking your medicine suddenly without your doctor's advice. Doing so could make your condition worse.

How to give this medicine to small children

In some special cases, this medicine can be given to babies as young as one month old. However, there are special precautions. That is, there are things to be very careful about. Therefore, talk to your doctor about this and follow his instructions exactly. The amount of medicine is determined according to the weight of the child.

Most importantly: This medicine is for you only. Do not give it to anyone else, even if they have the same symptoms as you. Everyone's condition is different, so it's not a good idea to give this medicine to anyone without first consulting a doctor.

What do you do if you miss a medication appointment?

Please, don't panic at such times. If you miss a dose, take it as soon as you remember. However, if it is almost time for your next dose (i.e., within a few hours), skip the missed dose and take the next dose at the scheduled time. Never take a double dose.You should only take the prescribed amount per day. If you are in doubt about this, ask your doctor or pharmacist.

What to do if you take too much? (Overdose)

If you think you have taken too much medicine, or if your child has accidentally taken too much, go to the nearest hospital emergency room immediately. Or call a poison control center. This should be treated as an emergency , do not delay.

Other medications that may interact with this medicine (Interactions)

There are some medications that may not work properly or cause other problems when taken with Sapropterin. We call these interactions. Therefore, tell your doctor about any medications you are taking.

• Avanafil `( Avanafil )`
• Levodopa (a medicine for Parkinson's disease)
• Methotrexate (used for diseases such as cancer and arthritis)
• Pemetrexed
• Pyrimethamine (a medicine used to treat malaria)
• Sildenafil `(Sildenafil)`
• Tadalafil `(Tadalafil)`
• Vardenafil `(Vardenafil)`

This list may not include all medications. Therefore, tell your doctor about all medications you take (including vitamins, herbal remedies, and dietary supplements). Also tell your doctor if you smoke, drink alcohol, or use illegal drugs. Some things can interact with your medications.

Things to be careful about when taking medicine

While you are taking this medicine, your doctor will monitor your condition closely. See your doctor regularly and have blood tests done regularly. These blood tests check your phenylalanine levels and see if the medicine is working properly. Remember, it can take up to 4 weeks for your body to respond to this medicine. So don't panic, just be patient.

If your symptoms do not improve, or if they get worse, tell your doctor right away.

The most important thing is that you need to follow a special diet while taking this medicine. You need to eat foods that are low in phenylalanine. This is essential for PKU. Before making any changes to your diet, be sure to talk to your doctor or a dietitian/nutritionist. They can give you a list of foods that are good for you and advice on how to prepare your meals.

Imagine, Nalini Akka, while giving this medicine to her daughter who has PKU, is controlling her diet as the doctor has told her. Before she prepares food, she always looks for foods that are low in phenylalanine. When she goes to the store, she reads the labels on food packets. That kind of care is needed in this situation.

What are the side effects?

Like any medicine, Sapropterin can cause side effects in some people. However, not everyone gets them. Let's take a look at what they are.

Side effects that you should tell your doctor immediately:

If you experience any of these symptoms, you should tell your doctor without delay. These can be serious.

• Allergic reactions: Skin rash, itching, redness of the skin, swelling of the face, lips, tongue or throat (this can also make it difficult to breathe).
• Hyperactivity: Restlessness, fidgeting, talking excessively, and difficulty paying attention.
• Inflammation of the esophagus or stomach: Loss of appetite, sore throat, pain or difficulty swallowing, burning in the chest, nausea, vomiting, dry cough, bloody or tarry stools.

Side effects that usually do not require medical advice, but should be reported to the doctor if they persist:

These usually subside in a few days. But if they persist, or if they bother you, tell your doctor.

• Cough
• Diarrhea
• Headache
• Runny nose or stuffy nose
• Sore throat
• Vomiting

This list may not include all side effects. If you notice anything else unusual, ask your doctor about it.

How do you store the medicine?

It is also very important to store this medicine properly.

• Keep it out of the reach of children and pets.
• Store at room temperature (between 20 and 25 degrees Celsius, between 68 and 77 degrees Fahrenheit). Do not expose to extreme heat or cold.
• Keep this medicine in the original container it came in.
• Keep the bottle tightly closed to prevent moisture from escaping.
• Do not use expired medication and dispose of it carefully. You can ask your pharmacy for instructions on how to dispose of it properly.

Finally, things to remember

Well, we've talked a lot about Sapropterin, haven't we? Living with a condition called PKU can be a challenge, but with medication like this and proper medical advice, you and your child can overcome that challenge.

"Remember, it is very important to take this medicine exactly as the doctor prescribed, in the amount prescribed, and at the time prescribed. Also, it is essential to follow a special diet.""If you do both of these things properly, you will be able to control your PKU condition well and live a healthy life."

If you have any questions, concerns, or doubts, don't keep them to yourself, but feel free to ask your doctor. They are there to help you. You are not alone on this journey, we are all with you!

Wishing you and your baby good health!

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