Risdiplam Liquid: New Hope for Spinal Muscular Atrophy (SMA)

Does your child or family member have Spinal Muscular Atrophy, or SMA for short? If so, your doctor may have told you about a medicine called Risdiplam. When you hear this name, you may be a little scared. You may have many questions in your mind, such as, "What kind of medicine is this, how is it given, what will happen?" We are going to answer all of these questions in a simple way that you can understand. Let's find out exactly what this medicine is.

Simply put, what is Risdiplam?

Risdiplam (brand name Evrysdi) is a medication used to treat a condition called Spinal Muscular Atrophy (SMA). It comes as a solution.

Now let's see what this actually means. Imagine that our muscles need a special type of protein to function properly and keep them strong. In a person with SMA, due to genetic reasons, the body produces very little of this protein. This is why the muscles gradually weaken and atrophy.

So this Risdiplam drug does a wonderful job . It helps our body make more of that special protein that is lacking. It's like someone helping a factory to get it running smoothly again when it's a little slow. When this protein is produced in excess in the body, it helps to slow down the rate of muscle weakness and control the worsening of symptoms.

Things you need to tell your doctor before starting medication

It is very important to talk to your doctor completely openly before giving this medicine to you or your child. If any of the following apply to you, be sure to mention it.

How exactly do I use this medicine?

This is a bit of a responsible thing to do. But don't worry, you'll get used to it after a while. It's best to give the medicine at the same time every day, just as the doctor prescribed.

• Preparation: Wash your hands thoroughly with soap before preparing the medicine and after giving it.
• Correct measurement: You will receive a special oral syringe with this medicine. Use the syringe to measure the exact amount your doctor tells you to take. Do not use any other spoon or cup to measure.
• Immediate administration: After drawing the medicine into the syringe, it should be administered to the patient within 5 minutes . Do not leave it longer than that.
• Before or after food?: This can be given with or without food. If it seems like an upset stomach, it is best to give it after a meal. If you are breastfeeding, you can give it before or after breastfeeding.
• Do not mix: Never mix this medicine with formula or any other type of milk.
• Give some water: After giving the medicine, give the person some water to drink. This will help us make sure that all the medicine has been swallowed.
• If it gets on your skin or eyes: If you accidentally get the medicine on your skin, wash the area thoroughly with soap and water. If it gets in your eyes, rinse thoroughly with water only.

When given to small children

This medicine is sometimes recommended even for newborns. However, only your doctor can decide on this. Therefore, you should be especially careful when giving it to young children.

What to do if you miss a dose of medication?

We don't always remember everything. So it's possible to forget to give a dose of medicine. If that happens, don't worry.

Imagine you were supposed to give the medicine at 8 am. You forgot it.

• If it is not more than 6 hours late: If you remember before 2 pm (that is, within 6 hours of the scheduled time), take the missed dose as soon as you remember.
• If it's more than 6 hours late: If you remember after 2 pm, don't give it that time . Skip it altogether and give it the next time as scheduled at 8 am the next day.

Can there be interactions with other medications?

Yes, some medications can interact with Risdiplam. The main one that has been identified so far is the diabetes drug Metformin .

But this list is not complete. So when you go to see your doctor, tell him about every medicine you or your child is taking. That means,

• Medicines prescribed by doctors
• Non-prescription medicines that can be purchased at the pharmacy
• Vitamins, nutritional supplements
• Sinhala medicine, Ayurvedic medicine
• If you smoke or drink alcohol, tell them about it too.

Then your doctor can safely plan your treatment.

Special things to be aware of when taking medication

Pregnancy and birth control

This is very important . There is a high risk of serious birth defects in the baby if you become pregnant while taking this medication.

• Before starting the medication, you must take a pregnancy test to confirm that you are not pregnant.
• You must use a highly effective form of birth control during the entire time you are taking the pill and for at least one month after stopping the pill.
• Talk openly with your doctor about the birth control method that is right for you.

Fertility

There is some concern that this medication may have an effect on fertility. If you have any concerns about having children in the future, don't hesitate to talk to your doctor about it.

What are the possible side effects of the medication?

Like any medicine, this medicine can cause some side effects. However, not everyone gets them. The table below clearly shows what these are.

This list may not include all side effects. If you notice anything unusual in addition to these, tell your doctor.

How to store the medicine?

It is also very important to store this medicine properly.

• Best way: Keeping it in the refrigerator is best. But never in the freezer. Don't let it freeze. Keep it in the original bottle it came in, away from light.
• Storage period: The medicine should be used within 64 days from the day it is mixed and dispensed by the pharmacy. After that, discard any remaining medicine.
• Room temperature: In an emergency, such as when traveling, you can keep it at room temperature (below 40 degrees Celsius) for up to 5 days. Those 5 days don't have to be consecutive. For example, you left it out for a day today, put it back in the fridge, left it out for another day a week later... After the 5 days are up, you can't leave it out again.
• Keep away from children: As with all medicines, keep this one out of the reach of small children and pets.

How do you dispose of leftover medicine?

Do not simply throw expired or unwanted medications in the trash or flush them down the toilet. Ask your doctor for advice.

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