Some diseases are very rare. And living with them is a big challenge. Mucopolysaccharidosis VI, or MPS VI or Maroteaux-Lamy syndrome, is one such rare, genetic disease. Galsulfase is a special treatment that brings relief to those suffering from this disease. Today we will talk about this very important treatment. If you, your child or someone you know has been prescribed this treatment, this article will be very useful for you.
What is Galsulfase?
Simply put, Galsulfase is an enzyme replacement therapy. Now let's see what that means.
Think of our body as a big factory. This factory has special workers to do different jobs. These workers are what we call enzymes . In MPS VI, a special enzyme that is needed to break down complex sugars in our body is either not produced by the body at birth or is produced very little.
So when this enzyme is lost, those sugars start accumulating inside the body's cells. This is why various organs like bones, heart, lungs, and eyes are damaged and symptoms appear.
This medicine, called Galsulfase, works by giving the body that enzyme that is lacking from outside. When this medicine enters the body, it breaks down the accumulated sugars and helps control symptoms. The brand name for this medicine is Naglazyme.
Important: This is not a cure for the disease. However, it provides significant relief by controlling symptoms and improving quality of life.
Things you need to tell your doctor before starting treatment
Before starting this treatment, it is very important to inform your doctor about your complete health history. Be sure to tell them about the following in particular:
| Information you need to provide | Why is that important? |
|---|---|
| Allergies | Tell your doctor if you have any allergies to gliclazide, other medications, foods, or hamster proteins. This is important because these substances are used in the manufacture of some medications. |
| Snoring or sleep apnea | Some people with MPS VI may experience sleep apnea. This medication may change this condition, so your doctor should be aware of this. |
| Fever or infection | If you currently have an infection, such as a cold or flu, please tell us about it before receiving treatment. |
| Pregnancy or breastfeeding | If you are pregnant, trying to become pregnant, or breastfeeding, be sure to inform your doctor before treatment. |
How is this treatment given?
This is not a medicine you can take home.
- An injection into a vein: This is a treatment that is given into a vein, like saline. In medicine, we call this an intravenous infusion .
- In a hospital or clinic: This treatment is given in a hospital or specialized clinic by a trained doctor or nursing staff.
- Duration: This treatment usually takes a few hours to administer, during which time you will be kept under medical supervision.
Is this suitable for children?
Yes, this treatment is also given to children over the age of 5. However, the dosage is determined with special care, considering factors such as the child's condition and weight. Therefore, if you are giving this treatment to your child, talk to your pediatrician about it.
What to do if you miss a dose?
It is very important to receive this treatment on the scheduled date and time for the success of this treatment. If for some reason you are unable to come for the treatment on the scheduled date, do not skip it. Call your doctor or hospital as soon as possible and arrange another date.
What if the dosage of the medicine increases (overdose)?
Since this is given in a hospital, under medical supervision, the chance of an overdose is very low. However, if you suspect that this has happened, inform the medical staff immediately. If necessary, they will contact the National Poisons Information Centre at the Colombo National Hospital for advice, or refer you to the Emergency Treatment Unit (ETU) .
What are the possible side effects of the medication?
As with any medication, some side effects can occur with galsulfase. Some of these can be serious, so it's important to be aware of them.
| Side effects that should be reported to the doctor immediately | |
|---|---|
| 🔴 Allergic reactions | Skin rash, itching, swelling of the face, lips or tongue. |
| 🔴 Breathing problems | Difficulty breathing, wheezing. |
| 🔴 Chest pain | Feeling of tightness or pain in the chest. |
| 🔴 Other serious symptoms | Vision changes, ear pain or infection, dizziness, fever or chills, high blood pressure. |
| Usually not serious (but tell your doctor if it persists) | |
| 🟢 Body pain (Aches and pains) | Pain in muscles and joints. |
| 🟢 Digestive system problems | Stomach ache, nausea, vomiting, diarrhea. |
| 🟢 Other minor features | Headache, fatigue. |
This list does not include all possible side effects. If you experience any other symptoms, please inform your doctor.
Other medications that may interact with this medicine
Some medications can interact with galsulfase. For example:
- Chloroquine
- Gentamicin
- Hydroxychloroquine
This is not a complete list. Therefore, tell your doctor about all the medications you are taking (prescribed, over-the-counter, vitamins, traditional medicines, Ayurvedic medicines). Also, inform him/her if you smoke or drink alcohol.
We know that living with such a rare condition is not easy. But modern treatments like these can help you manage your symptoms and live a better life. The most important thing is to maintain a regular relationship with your doctor and follow their advice exactly.
Take-Home Message
- Galsulfase is an enzyme replacement therapy that controls the symptoms of a rare disease called MPS VI. It does not completely cure the disease.
- This treatment is given in a hospital or clinic, through a vein. It cannot be taken home and used.
- Before starting treatment, it is essential to inform your doctor about all allergies you have, other medical conditions, and all medications you are taking.
- If you experience serious allergic symptoms such as difficulty breathing and facial swelling, seek medical advice immediately.
- Do not miss your scheduled treatment date. If you do, call your doctor immediately.
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