Treatment

Which Treatment Should We Choose?

The advantages and disadvantages, and why my parents chose treatment with Zolgensma.

Mum would like to explain why she and Dad decided on this particular type of treatment.

Perhaps this post will be helpful to some parents facing the same situation. My parents were also interested in why the parents of other children with SMA had chosen particular treatments. The doctors offered to put them in contact with a very kind family whose children also have SMA. They could ask them questions that might help them choose between the treatments described below. The family agreed, and we are incredibly grateful to them for that. Their answers provided us with the information we needed.

One important thing to point out: no one will tell you or dictate to you (neither families who have been through this nor doctors) that you must choose this or that treatment because of a particular reason! However, you can get answers to your questions about why they chose a particular type of treatment, what advantages that treatment has had for them, and what the course of treatment was like after the medicine was administered. All this information was a great help to Mum and Dad. It allowed them to weigh up all the PROS AND CONS and make the BEST POSSIBLE DECISION FOR MY LIFE. In the case of Zolgensma, for example, the decision is irreversible.

There are currently three medicines available for SMA that can stop the progression of the disease. In other words, they cannot cure the condition, but they can halt its progression.

However, there are also many questions and uncertainties. Only five-year study data are available for treatment with Zolgensma. Once the medicine has been administered into a vein, it cannot be taken back. It can never be “removed” from the body. Among other things, the medicine may not be suitable for every child. It can cause numerous side effects (including damage to the liver and effects on the immune system), so patients must have suitable test results to be eligible for treatment.

ZOLGENSMA HAS ONE MAJOR LIMITATION. It can only be administered up to a certain age — before the child turns two — and up to a certain weight (13.5 kg). You can find more detailed information here:

https://smaci.cz/lecba-a-pece/zolgensma

Here is a brief overview of the treatment options available for SMA.

So why did Mum and Dad decide on Zolgensma?

After receiving all the explanations and information from the doctors, discussing everything with our family, and reading numerous studies in both Czech and English, Dad and the family decided on Zolgensma.

Fortunately, I was diagnosed before the disease had fully manifested itself. It was detected at a point when I was not showing the typical symptoms. Most importantly, Mum discovered the disease while I was still eligible to receive this rare medicine (I was 10 months old).

ZOLGENSMA HAS ONE MAJOR LIMITATION. You could compare it to a race against time when it comes to Zolgensma. The medicine can only be administered up to a certain age — before the child turns two — and up to a certain weight (13.5 kg).

Thank God, I won that race — and I won it by a mile! The medicine was administered in time, but I will have to live with the disease and its consequences for the rest of my life.

E.