New hope for Duchenne Muscular Dystrophy (DMD): Let's learn about the drug Duvyzat?

I understand how heavy and scary it must be when your doctor tells you that your child has Duchenne muscular dystrophy (DMD). This is a genetic disease that mainly affects boys and gradually weakens their muscles. Symptoms usually appear in early childhood. There is no complete cure for DMD yet. But don't worry. Medical science is advancing day by day. With that advancement, a new hope is this new drug called Duvyzat. Let's talk about it simply.

What is Duvyzat? How does it work?

Simply put, our muscles need constant maintenance to stay strong. Think of the muscles in our body as being like a brick wall. To keep this wall strong and bonded together, it needs a good layer of mortar. Similarly, to keep our muscle cells strong, a special protein called dystrophin , which is like mortar, is essential.

Duchenne muscular dystrophy (DMD) is a genetic condition that occurs when the body either doesn't produce enough of the dystrophin protein, doesn't produce enough of it, or doesn't produce enough of it. When this 'glue' is lost, the muscle wall becomes weak, breaks down and can't be repaired. That's why muscles weaken over time.

In addition to this problem, children with DMD have overactive enzymes called histone deacetylases (HDACs) . This causes chronic inflammation in the muscles. This further reduces muscle repair, and muscle is replaced by fat and scar tissue.

Until now, doctors have used steroids, physical therapy, and assistive devices like wheelchairs to treat DMD. But Duvyzat is the first nonsteroidal drug. It works by controlling the overactive HDAC enzyme. This reduces inflammation in the muscles and allows them to break down. This means that the speed at which muscles weaken is slowed.

How should I use this medicine?

Duvyzat is a liquid medicine that you take by mouth. It should be taken twice a day, in the morning and at night, with food .

• It is very important to take the medicine at the same time every day .
• Before using the medicine bottle, you should shake it well for at least 30 seconds .
• Measure the medicine, using the special syringe that comes with it.
• Most importantly: Do not mix this medicine with water or any other liquid.

Your doctor may give this medicine alone, or he may give it along with steroids. It will be decided based on the child's condition.

How have the properties of this medicine been studied?

In a major study to examine the effectiveness and safety of this drug, boys with DMD were divided into two groups.

1. First group: They were given the drug Duvyzat.

2. Group 2: They were given something that looked like medicine, but had no active ingredient in it. In medicine, we call this a 'placebo' .

This is done to measure the true effect of the drug, how different it is from just being there. All the participants in this study were boys. Their average age was 9 years old.

After giving these treatments for 72 weeks, the changes in the children's abilities were measured. Several other tests were used for this.

• North Star Ambulatory Assessment (NSAA) score: A method of measuring the ability of someone with DMD to perform daily activities.
• Six-minute walk test: Sees how far you can walk in 6 minutes.
• MRI tests: Measures changes in fat deposits in the muscles of the thigh area.

What were the results of the study?

The results are really encouraging. DMD is a condition where symptoms get worse over time. What was clear in this study was that the rate of deterioration in the children who took Duvyzat was much slower than in the children who didn't take the drug (the placebo) .

• Strength and mobility: The time it took to climb four flights of stairs increased by only 1.25 seconds after 72 weeks in the children who took Duvyzat. But it took about 3 seconds longer in the children who didn't take the drug. That means Duvyzat slowed the decline in their ability to climb stairs by about 40%.
• NSAA scores: At the end of the study, the decrease in NSAA scores was significantly less in children who took Duvyzat than in those who did not take the drug. This also indicates that the rate of muscle strength decline was reduced.

These results may vary for your child, so you should discuss the benefits and risks of this medication with your doctor before making a decision.

Side effects and how to manage them?

Like any medication, Duvyzat can have side effects. It is important to be aware of this.

These are not the only symptoms. If you experience any other unusual symptoms, talk to your doctor about them.

Can there be problems with other medications?

Yes, Duvyzat can interact with some other medications. Therefore, it is important to tell your doctor about all the medications your child is taking, including prescription medications, over-the-counter (OTC) medications, vitamins, and herbal products.

In particular, inform your doctor if you are taking the following medications:

• Some cholesterol-lowering medications (e.g. atorvastatin, simvastatin)
• Diabetes medications like metformin
• Medications that affect heart rhythm (e.g. amiodarone)
• Some antibiotics (e.g. azithromycin, ciprofloxacin)
• Some medications for mental illness
• Some medicines for allergies

This list is not complete, so it's best to tell your doctor about everything your child is taking.

Duvyzat, Duchenne Muscular Dystrophy, DMD, muscular dystrophy, dystrophin, pediatrics, genetic diseases, new drugs