Avalglucosidase Alfa (Nexviazyme) - Everything about this drug for Pompe disease in simple terms

Has your doctor told you or someone in your family has a rare condition called Pompe disease? If so, we're going to talk about a medication for it today. The name of this medication is Avalglucosidase Alfa. You may know it by the brand name Nexviazyme. This name is a bit complicated, so you may have a lot of questions about it. Don't worry, we'll talk about all of this in a very simple way that you can understand.

Simply put, what kind of medicine is this?

Imagine that our body is like a big factory. For everything to work properly in this factory, there must be workers doing various jobs. We call these workers in our body enzymes .

People with Pompe disease are born with a deficiency of a specific enzyme. This enzyme's job is to break down and control the sugar called glycogen in our muscles and cells. When this enzyme is missing, the sugar called glycogen starts to build up in our muscles. It's like garbage in a factory. When this builds up, the muscles become weak, they have difficulty breathing, and the heart is affected.

So, what this drug called Avalglucosidase Alfa does is replace the function of that missing, reduced enzyme. That is, it's like bringing in someone from outside to do the same job instead of the reduced employee. When this drug enters the body through a vein, it starts to break down that accumulated glycogen and remove it. Then the symptoms gradually decrease, and you start to feel relief.

Things you need to tell your doctor before taking this medicine

Before starting this medication, it is very important that your doctor is aware of your complete health condition. This is so that you can be treated more safely. Be sure to tell them about these things in particular.

• If you have heart disease .
• If you have a lung disease .
• If you have previously had an allergic reaction to this Avalglucosidase Alfa medicine, any other medicine, food, dye, or preservative.
• If you are pregnant or trying to get pregnant.
• If you are a breastfeeding mother.

How to use the medicine?

This is not a medicine you can use at home. It is given under medical supervision in a hospital or clinic.

• A drug given through a vein: This drug is given into a vein, like saline. We call this process an infusion .
• Administered by a medical team: This medicine is given to you by a trained doctor or nursing staff, so there is nothing to worry about.

Can this medicine be given to small children?

Yes, sometimes this medicine is given to children as young as one year old. But it is done under special medical advice and precautions. If you are giving this medicine to your child, talk to your medical team about it and explain everything.

What do you do if the dose increases?

Since this is a hospital-administered medication, the chances of an overdose are very low. But if you think you may have somehow overdosed, don't panic. There are two things you should do immediately.

1. Go to the hospital's Emergency Treatment Unit (ETU) immediately.

2. Alternatively, call the National Poison Information Centre at the Colombo National Hospital .

This is a very urgent situation, so it is important to act quickly.

What if I miss a dose of medicine?

Taking this medicine on time and on the prescribed date is essential for the success of the treatment. If you are unable to go to the hospital on the prescribed day, do not miss it. Call your medical team immediately and let them know. They will tell you what to do next.

Special points to be aware of

• Medical checkups: Don't miss out on regular checkups at the clinics. Talk to your doctor about whether your symptoms are improving, worsening, or if you have any new problems.
• Allergic Reactions: Some people may have a severe allergic reaction to this medicine or shortly after it is given to them. To reduce this risk, your healthcare team may give you other medications (such as piritan) before you start taking it. So follow their instructions exactly.

What are the possible side effects of this medicine?

Like any medicine, this can cause side effects in some people. But not everyone gets them. The important thing is to know what those side effects are and what to do if they occur.

Can there be problems with other medications?

According to current information, this medicine has not been reported to cause major problems with other medications. However, give your doctor a complete list of all the medications you are taking (including vitamins, herbal remedies, and supplements). Also, tell your doctor if you smoke or drink alcohol. This can help prevent unnecessary problems.

Pompe disease, Avalglucosidase Alfa, Nexviazyme, enzyme therapy, genetic diseases, muscle wasting, glycogen