Have you heard of a condition in your family, especially a young child, where the muscles in the spine gradually weaken? Medically, we call this Spinal Muscular Atrophy (SMA) . In Sinhala, it is a degeneration of the muscles associated with the spine. This is a genetic disease that is passed down from generation to generation. This is why this muscle weakness can affect not only the tasks we usually do every day, such as holding our head up straight, sitting without help, walking, swallowing food, but also breathing. So, a new oral medication that is given as a treatment for this SMA disease is called Evrysdi (risdiplam) . Today, we will talk about this medicine, how to use it, what are its benefits, and what are the side effects that you should know about.
A brief introduction to SMA disease and its variants
Before we talk about the Evrysdi drug, let's understand a little about SMA. The disease is divided into several main types based on the age at which symptoms begin to appear.
- Infantile-onset SMA (Type 1): This is the most severe type. Symptoms usually appear in infancy, before the age of 6 months.
- Later-onset SMA (Type 2 and 3): In this type, symptoms begin to appear in childhood or adolescence.
- Adult-onset SMA (Type 4): In this case, symptoms only appear after adulthood.
The drug Evrysdi is used to treat all of these types. It is also given to people who have been diagnosed with SMA through genetic testing but are not yet symptomatic (pre-symptomatic).
How to use Evrysdi medicine?
This is a very important part, because using the medicine correctly directly affects the results .
You will receive this medication from a specialty pharmacy, in liquid form. If you ever receive a bottle that contains something that looks like a powder, do not mix it up and drink it. If this happens, immediately contact the place where you were given the medication or your doctor.
How to prepare the medicine
Your doctor will tell you exactly how much to take and how to measure and prepare it. The bottle of medicine should come with a cap, a bottle adapter, and several reusable syringes. If any of these items are missing or broken, talk to your doctor before taking the medicine.
- It is important to take the medicine at the same time every day .
- The best time to take the medicine is after a meal . If it's a baby, give it after breastfeeding is finished.
- Never mix this medicine with formula or any other type of milk, such as cow's milk.
- After taking the medicine, drink some water to make sure it is completely swallowed.
- Some people are given this medicine through a feeding tube. In that case, you may need to flush the tube with water after giving the medicine.
Most importantly: Use the special syringe you were given to measure the dose. If it is lost or broken, ask your doctor before getting a new one. The used syringe can be washed, cleaned, and used the next day.
Things to be careful about when taking medicine
After drawing up the dose of medicine into the syringe, you should drink it within 5 minutes . If you are unable to drink it within that time, discard the dose and prepare a new dose.
Also, do not let this medicine get on your skin or in your eyes. If it gets on your skin, wash it off thoroughly with soap and water. If it gets in your eyes, rinse your eyes thoroughly with clean water. Do not mix two bottles of medicine for one dose for any reason.
How was the success of this medicine tested?
Before any medicine comes to the market, many clinical studies are conducted to find out how effective and safe it is. The Evrysdi medicine has also undergone several such tests. Let's take a simple look at it.
| Test type | Those who participated in the test | The main results observed |
|---|---|---|
| Infantile-onset SMA | 58 infants, approximately 6 months old, with Type 1 SMA. | After 12 months of taking the drug, about a third were able to sit for 5 seconds without help. After 24 months, about 60% were able to sit that way. |
| Later-onset SMA | 180 children and adults aged 2-25 years with type 2 or 3 SMA. (Some were given Evrysdi, some were given a placebo.) | Motor function improved in those who took Evrysdi, while it declined in those who took the placebo. |
| Those who have not shown symptoms (Pre-symptomatic) | 26 babies under 6 weeks old who were diagnosed with SMA through genetic testing, but were asymptomatic. | At the end of 12 months, 96% were able to sit for 5 seconds without help. 81% were able to sit for 30 seconds. |
Remember, these are research results. The results you or your child will get may be different. It varies depending on each person's physical condition.
What are the side effects of Evrysdi?
As with any medicine, Evrysdi can cause some minor side effects. The most common are:
- Fever
- Diarrhea
- Skin rash or itching
These are the most common side effects, but there are also other possible side effects.
What to do if side effects occur?
In cases like fever, you can talk to your doctor and get an appropriate dose of paracetamol.
If you have a loose stomach, the most important thing is to prevent dehydration.
- Give them plenty of fluids to drink.
- If it's a baby, breastfeed frequently.
- It is very good to give young children a drink like Jeevani (oral rehydrating solution) .
- Drink plenty of water.
The most important thing is to immediately inform your doctor about any symptoms that bother you or seem unusual. Don't just self-medicate.
Is Evrysdi safe during pregnancy and breastfeeding?
This is a very important matter.
- If you are pregnant, planning to become pregnant, or are breastfeeding, be sure to tell your doctor before starting Evrysdi.
- The current opinion is that the Evrysdi drug may be harmful to the baby in the womb.
- Therefore, before starting this medication, you should take a pregnancy test to make sure you are not pregnant.
- You must use a highly effective form of birth control while taking the pill and for at least one month after stopping the pill.
Feel free to discuss any questions or concerns you have about this with your doctor.
Take-Home Message
- Spinal Muscular Atrophy (SMA) is a genetic disease that causes muscle weakness. Evrysdi is an oral liquid medicine for this condition.
- You should take the medicine exactly as your doctor tells you, at the same time every day, preferably after meals.
- Do not mix the medicine with dairy products. Drink some water after taking the medicine.
- Side effects such as fever, stomach ache, and skin rashes may occur. Tell your doctor about any bothersome symptoms.
- If you are pregnant, trying to become pregnant, or breastfeeding, be sure to discuss taking this medication with your doctor.
- Talk openly with your doctor about any questions or concerns you may have. That's the best place to get accurate information.
💬 අදහස් (0)
තවමත් කිසිදු අදහසක් පළ කර නොමැත. ඔබේ අදහස පළමු වරට මෙහි එක් කරන්න.
ඔබේ අදහස එක් කරන්න