Worried about your child's growth due to achondroplasia? Let's learn about the drug Voxzogo (vosoritide)

You may be worried, perhaps even scared, about your child's growth, especially their height. It's normal to feel this way as a parent living with a condition called Achondroplasia. But you're not alone. With advances in medical science, there are now new treatments that can help with these conditions. Today, we're talking about a drug called Voxzogo, which has brought such new hope, specifically for children with Achondroplasia.

What exactly is achondroplasia?

Simply put, Achondroplasia is a genetic condition that causes dwarfism. It affects the way children's bones grow, especially the long bones (those in the arms and legs). It is a very rare condition, occurring in about one in 15,000 to 40,000 births.

In most cases, this is not something that is inherited from the parents, but something that occurs in the child's own body due to a new genetic change (mutation). So don't feel guilty about this. This condition can cause additional health problems as the child grows up, such as obesity, ear infections, and problems with the nerves in the spine and brain.

There are several common symptoms that can be seen in a child with achondroplasia.

How does Voxzogo work?

To understand this, we first need to learn a little about growth plates . Think of these growth plates at the ends of our long bones as the space where new asphalt is constantly being laid when building a road. They are made of a strong, flexible tissue called cartilage. As a child grows, this cartilage turns into bone, and the bone lengthens. When the child is fully grown, these growth plates close, and height growth stops.

In children with achondroplasia, a mutation in the genes (DNA) disrupts one of the body's message-transmitting systems. There are two message systems in our body that affect bone growth:

1. System that increases bone growth.

2. The system that reduces (stops) bone growth.

In achondroplasia, the genetic change causes the signaling system that slows down bone growth to become overactive.

The goal of this treatment is to use this medication while the child's growth plates are open, helping them achieve maximum growth. When the growth plates close, meaning the child stops growing, the medication is stopped. This is determined by your doctor.

How do you use this medicine?

Voxzogo is a vaccine that is given to your child under the skin every day, at about the same time each day . It is designed so that you can give it at home.

• Injection sites: This is injected under your child's skin. You can use your child's thigh, stomach (about two inches from the navel), buttocks, or the back of their upper arm .
• The most important thing: Don't keep injecting in the same place. If you inject in one place today, inject in a different place tomorrow. We call this `injection site rotation`. This can reduce discomfort such as redness and pain. Do not inject into areas with swollen, bruised, red, or hard skin.
• A must-do: One hour before the injection, feed your child a good meal and give him a large glass (about 8-10 ounces) of water, milk, or fruit juice. This medicine can sometimes cause low blood pressure, which can make you feel tired or dizzy. Giving your child food and fluids beforehand can greatly reduce this risk.

The dose of medicine given to a child is determined by the child's weight. You should tell the doctor if the child's weight changes. Your doctor will train you on how to inject this into your child exactly. Never try to do this yourself until you have received that training. Also read the instruction leaflet that comes with the medicine carefully.

What are the possible side effects?

Like any medication, Voxzogo can cause some side effects. It is important to be aware of these.

• Injection site reactions: This is the most common side effect. There may be redness, swelling, itching, bruising, or pain at the injection site. This can be treated by applying a cool compress to the area. Be sure to talk to your doctor before taking any medication for pain or itching.
• Low Blood Pressure: As we mentioned earlier, this is best avoided by giving food and drink before the injection. If your child feels dizzy or tired, tell them to get up slowly, using a support if necessary. If they feel dizzy, tell them to sit or lie down. If this condition persists or worsens, seek medical advice.
• Stomach upset and vomiting: Some children may have a stomach upset (gastroenteritis) or vomiting. During this time, give them plenty of fluids to drink and small amounts of bland, light foods (e.g., crackers, toast, bananas, light soup). If these persist, tell your doctor.
• Joint pain: Some children may experience joint pain. Talk to your doctor about this as well.
• Changes in blood tests: This medication may increase the level of a blood test called alkaline phosphatase. This is a measure of liver or bone function. Therefore, it is very important to continue to have blood tests as prescribed by your doctor.

A few more important things to know

How long does it take for this medicine to work?

According to research, it takes several months to see an increase in the growth rate of children. However, the results may vary from child to child. So be patient and continue the treatment as the doctor says. The doctor will constantly monitor the child's progress.

Who is this medicine not suitable for?

If your child has any kidney-related conditions, be sure to inform the doctor. The doctor may decide not to prescribe this medicine to children with some severe kidney conditions.

Is this a special medicine?

Yes. Voxzogo is a specialty medicine . This means that it is not a medicine that you can get at a regular pharmacy. These are expensive medicines for rare or complex diseases. There is a special procedure for obtaining these. After your doctor prescribes this medicine, a specialty pharmacy will contact you and explain how to get the medicine delivered to your home.

Achondroplasia, Voxzogo, vosoritide, pediatrics, height loss, genetic diseases, growth and development