New hope for those with Friedreich Ataxia: Let's learn about the Skyclarys drug!

Friedreich Ataxia is a rare, hereditary condition that primarily affects our nervous system. Although symptoms can appear at a young age, we know how challenging it is to live with it. It is difficult to put into words the sadness and anxiety that comes with gradually losing control of our bodies. However, with the advancement of science, we are getting new hope to deal with such diseases. That is the kind of new hope that we are talking about today.

First, let's see what Friedreich Ataxia is.

Simply put, Friedreich's ataxia (FA) is a genetic disease that damages our nervous system. This gradually reduces the ability to control our muscles. As the disease progresses, the symptoms can get worse.

Let's take a look at what symptoms are commonly seen.

What is this new drug called Skyclarys?

Skyclarys is the first treatment approved for Friedreich's ataxia (FA) in 2023. It contains the active ingredient `omaveloxolone`.

Studies have shown that Skyclarys can improve nerve function and slow the progression of the disease.

How does this medicine affect the body?

Although this is a bit of a scientific matter, let's understand it simply. There is a protein called `Nrf2` that protects the cells in our body. Think of it like a defense system in our body. In a person with FA, this `Nrf2` defense system does not work properly. Therefore, unnecessary stress and inflammation occur inside the cells, which damages the cells.

Skyclarys works by reactivating the weakened `Nrf2` defense system. That is, it "powers" it up. As a result, the mitochondria, which are like power plants that provide energy to cells, begin to function normally. This reduces the damage to cells.

How should I use the medicine?

Skyclarys is an oral capsule. You should be aware of these things when using it.

• You have to take three 50-milligram capsules a day at the same time.
• This should be taken on an empty stomach , that is, at least an hour before eating.
• The capsule should be swallowed whole . It should not be crushed or chewed.
• If you suddenly have difficulty swallowing the capsule, you can carefully open the capsule, add the powdered medicine inside to two tablespoons of applesauce, mix well, and swallow it all at once.
• Never mix this medicine with milk or orange juice.
• Also, you cannot open the capsule and give the medicine to someone who is receiving food through an enteral feeding tube.
• If you miss a dose, skip that dose and take your next dose at the scheduled time. Do not take a double dose to make up for a missed dose.

Side effects and how to manage them?

Like any medicine, Skyclarys can cause side effects . The most common side effects are:

• Increased liver enzymes
• Headache
• Nausea
• Stomach pain
• Fatigue
• Diarrhea

Of these, you should be especially concerned about elevated liver enzymes . This means that your liver may be under some strain or damage. For this reason, your doctor will often check for this by doing bloodwork. If you experience any of the symptoms below, tell your doctor right away.

• Nausea or vomiting
• Yellowing of the skin or eyes
• Pain in the upper right side of the abdomen

Things like these can help manage other side effects:

• For headaches: Drink plenty of water, eat nutritious foods, and get enough sleep. When a headache strikes, rest in a cool, dark room.
• Nausea, stomach pain, and diarrhea: Eat small meals rather than large meals. Eat slowly and chew your food thoroughly. Avoid caffeine, alcohol, and carbonated drinks. If you have diarrhea, you should stay hydrated. Drink water, soups, and orange juice are good. Eat bland foods like rice, potatoes, bread, and bananas. If your diarrhea is severe, talk to your doctor about appropriate treatment.
• Fatigue: Avoid driving or doing anything that requires alertness until you know exactly how this medicine affects you.

Can there be problems with other medications?

Yes, this is a very important point. Skyclarys can interact with other medications.

• Other medications: Some medications interact with an enzyme in our body called `CYP3A4`. If you are taking such a medication, your doctor may need to change the dose of Skyclarys or switch you to another medication. Therefore, tell your doctor about all the medications you are taking (including vitamins and herbal products).
• Birth control pills: This is very important. Hormonal birth control pills may be less effective when taken with Skyclarys. This means that there is a risk of pregnancy. Therefore, you should use a non-hormonal form of birth control, such as condoms, while taking Skyclarys and for 28 days after stopping the pill.
• Grapefruit and Grapefruit Juice: Avoid drinking grapefruit or grapefruit juice while taking Skyclarys. These can increase the effects of the medication and increase side effects.

Ultimately, you and your neurologist should decide together whether this medication is right for you. This is a prescription-only medication, available only at specialty pharmacies. Your doctor will make the necessary arrangements.

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