Fabry disease is a rare, genetic condition that occurs due to the deficiency of a very important enzyme in our body. This can affect various parts of the body, such as the heart, kidneys, and nervous system, over time. So, if you also have Fabry disease, your doctor may have prescribed you a medicine called Agalsidase Beta. Today, we will talk about this medicine in a simple and clear way.
What kind of medicine is this?
Simply put, Agalsidase Beta (brand name Fabrazyme) is a treatment that replaces the deficient enzyme in the body of a person with Fabry disease. In medical terms, this is called 'Enzyme Replacement Therapy' (ERT).
The most important thing is that this is not a cure for the disease. However, this treatment can greatly help control the damage caused by the disease to the body and reduce symptoms.
The most important things to tell your doctor
Before starting this treatment, it is essential that the doctor knows certain things about your health condition. Therefore, if you have any of the following, be sure to tell the doctor about it.
- If you have heart disease .
- If you have ever had an allergic reaction to Agalsidase Beta, mannitol, or any other medicine, food, or dye.
- If you are pregnant , or are expecting to become pregnant soon.
- If you are a mother who is breastfeeding your child .
How do you use this medicine?
This medicine is given as an intravenous infusion (infusion) into a vein. This is not something you can do at home. This treatment is only given by a trained health care provider in a hospital or clinic, under medical supervision.
Use for young children
Special care should be taken when giving this medicine to young children. Sometimes it is given to children as young as 2 years old, but only the doctor who examines the child makes the decision.
What if I take too much medicine?
Since this medicine is given in a hospital, under the supervision of a professional, the chances of an overdose are usually very low. However, if you suspect that something has happened, immediately call the National Poisons Information Centre at the Colombo National Hospital , or go to the nearest Emergency Treatment Unit (ETU) .
What to do if you miss a dose of medication?
It is very important to take the medicine on the scheduled day and at the scheduled time for this treatment. If for some reason you are unable to come to the hospital on the scheduled day, inform your doctor or clinic in advance and arrange another date.
Where do you keep the medicine?
Since this medicine is given in a hospital or clinic, you will not be allowed to take it home or keep it.
Other medications that may interact with this medicine
Some of the medicines you are taking may interact with Agalsidase Beta. Therefore, it is very important to tell your doctor about all the medicines you are taking (prescribed, over-the-counter, herbal products, vitamins). Especially if you are taking any of the following medicines:
- amiodarone
- chloroquine
- gentamicin
- Hydroxychloroquine
- monobenzone
This list does not include all possible drug interactions, so it is safest to inform your doctor about everything you are taking.
What are the possible side effects?
Like any medicine, this one can have some side effects. Some are serious, some are common. It's important to be aware of these.
| Side effect | Description |
|---|---|
| Side effects that should be reported to the doctor or nurse immediately | |
| Severe Allergic Reactions | Skin rash, itching, swelling of the face, lips or tongue. |
| Difficulty breathing | Shortness of breath, rapid breathing. |
| Chest pain or tightness | Feeling like your heart is being squeezed. |
| Heart palpitations | Feeling like your heart is beating fast or your heart rate is changing. |
| Other | Severe dizziness, swelling of the hands or feet, depression. |
| Side effects that are usually not serious (but should be reported to the doctor if they persist) | |
| What you feel when you inject the medicine | Fever or feeling cold. |
| General pain | Body aches, headaches. |
| Digestive system problems | Nausea, vomiting, stomach pain or discomfort. |
| Mental state | Feeling uneasy, anxious. |
This list does not include all possible side effects. If you experience anything different, talk to your doctor about it.
Take-Home Message
- Agalsidase Beta (Fabrazyme) is an enzyme replacement therapy for Fabry disease. Although it does not cure the disease completely, it can greatly help control symptoms and complications.
- This medicine is given intravenously in a hospital, so it is very important to receive treatment on time.
- You should tell your doctor about any other medications you are taking, any allergies you have, and if you are pregnant or breastfeeding.
- If you experience any symptoms such as difficulty breathing, chest pain, or skin rash during or shortly after injecting the medicine, tell your doctor or nurse immediately.
- If you have any questions about this medicine, don't be afraid or hesitant to ask your doctor.
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