If someone in your family, perhaps your own child, suffers from a condition called Duchenne muscular dystrophy (DMD), you are probably constantly searching for treatments for it. This is a genetic condition that gradually weakens the muscles. So, a new treatment for this is called Golodirsen. In this article, we will talk about this drug in a simple way that you can understand.
What is this medicine called Golodirsen?
Simply put, this is a drug that is used to treat Duchenne Muscular Dystrophy (DMD). Now let's see how it works.
Imagine that the muscles in our body are like a wall. The bricks in this wall are held together by a protein called 'dystrophin', like mortar. In a person with DMD, this mortar, that is, the dystrophin protein, is not produced. So the wall called muscle is not strong, and gradually weakens.
The drug Golodirsen works by helping the body make this dystrophin protein. This helps to increase muscle strength and control the progression of the disease.
Things you should tell your doctor before taking medication
Before starting this treatment, it is very important to give the doctor a complete description of your or your child's health condition. Be sure to mention these things in particular.
- Kidney disease: If you have any kidney-related condition, you should definitely tell us about it.
- Allergies: Tell your doctor if you have any allergies to Golodirsan or any other medicine, food, or dye.
- If you are pregnant or trying to get pregnant: If you are pregnant or planning to have a baby, tell your doctor.
- If you are a breastfeeding mother: If you are a breastfeeding mother, you should also mention that.
Based on this information, the doctor will decide whether this medicine is suitable for you or not.
How do you use this medicine?
This is not a medicine you can take home and use.
This medicine is given as an injection into a vein . It is given only by a doctor or a trained nurse, in a hospital or clinic .
So you don't need to worry about the dosage or the way it is given. The medical team will take care of those things.
Can this medicine be given to children?
Yes, this medicine is also given to children with DMD. However, your doctor will explain to you the precautions and special considerations involved.
What if I take too much medicine?
This is given in a hospital setting, so the chances of this happening are very low. However, if you think you may have taken too much of the medicine, immediately call the National Poisons Information Centre at the Colombo National Hospital , or go to the Emergency Treatment Unit (ETU) of the nearest hospital.
Important: This medicine is for the patient only. Never give this medicine to anyone else.
What do you do if you miss a dose of medication?
This is a medication that needs to be taken continuously, on a set schedule. Therefore, it is very important to follow the days and times that your doctor gives you exactly. If for some reason you cannot go on the scheduled date, call your doctor or clinic immediately and let them know. They will tell you what to do next. Do not skip your medication for any reason.
What are the possible side effects?
Like any medicine, the Golodirsan vaccine can cause some side effects. But not everyone gets them. Let's break these down into two parts.
| Possible side effects of Golodirsen | |
|---|---|
| Serious side effects that require immediate medical attention | |
| Allergic reactions | Symptoms such as skin rash, itching, redness, swelling of the face, lips, tongue, or throat. |
| Kidney injury | Decreased urine output, swelling of the ankles, hands, or feet. |
| Side effects that are usually not serious, but should be reported to the doctor if they persist | |
| Headache | Frequent headaches. |
| Fever | Feeling feverish. |
| Cough and cold | Cough, runny nose, or stuffy nose. |
| Digestive system problems | Stomach ache, nausea, vomiting. |
This list does not include all possible side effects. If you experience anything different, talk to your doctor about it.
Take-Home Message
- Golodirsen is a special vaccine used to treat Duchenne muscular dystrophy (DMD).
- This helps the body make a protein called dystrophin, which is essential for muscles.
- This medicine is given intravenously in a hospital or clinic. Therefore, you will not be allowed to take it home.
- Before starting treatment, tell your doctor about all of your medical conditions, especially kidney disease and allergies.
- If you experience any allergic symptoms such as rash, swelling, or symptoms that suggest kidney damage, such as decreased urine output, notify your doctor immediately .
- Be sure to go to the doctor on the days and times he gives you and get treatment. Don't skip your medication appointments.
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