"Lost Window." Administering a $1.8 million injection to Ksyusha from the orphanage is currently impossible

Ksyusha Maiseienka

Ksyusha Maiseienka from Mogilev is three and a half years old. In 2024, the girl was diagnosed with type two spinal muscular atrophy (SMA). This is a rare genetic disease in which neurons responsible for movement gradually die.

Muscles become progressively weaker. The child loses strength, first in the arms and legs, then it becomes difficult to sit, hold her head, swallow food, and breathe. And then — death.

There are medicines in the world that can stop the disease. This is Zolgensma gene therapy, arguably the most expensive medicine in the world. A child is literally cured after a single injection. The problem is that its cost is about $1.8 million. And in Belarus, this treatment is not included in the free healthcare program.

The peculiarity of the little girl's situation is that she lives in a Mogilev orphanage. Her father committed suicide. And her mother couldn't cope with the burden of the situation and gave the girl up.

Effectively, there was no one to fight for Ksyusha — until volunteers learned about the seriously ill girl and launched an urgent fundraiser.

As a result, a colossal sum was raised in record time — within a month. No similar fundraiser in the history of Belarus has ever happened so quickly.

Now many are concerned about when Ksyusha will receive that life-saving injection. But the answer is sad — unfortunately, at this moment it is impossible. And it is unclear if it will ever be possible in the future.

A republican medical council was held at the RSPC "Mother and Child". The official conclusion states that Ksyusha currently cannot stand or sit independently (according to her companions, she used to be able to sit), does not crawl, cannot lift her head or hold it when lying on her stomach.

It is worth noting that before the council, Ksyusha was admitted to the hospital with pneumonia — the illness became an additional burden on her body.

Zolgensma gene therapy should be administered as early as possible — while the child retains as many motor skills as possible. If Ksyusha's mother had started fighting for medication for her daughter immediately after the diagnosis became known in 2024, there would have been more opportunities. But she did almost nothing, and as a result, the girl's condition relentlessly worsened for two years.

Belarusian doctors assessed Ksyusha's condition and unanimously decided that her window of opportunity has been lost. At this moment, administering gene therapy carries more danger than potential benefit. The council's decision: gene replacement therapy is currently not indicated for the patient.

In this situation, for Ksyusha, in addition to various examinations, rehabilitations, and daily therapeutic exercises, it is also recommended to continue pathogenetic therapy with "Risdiplam" (taking into account that the girl's representatives have three vials).

"Risdiplam" is a drug that slows down the worsening of the condition and can somewhat improve motor functions and support breathing. It does not cure SMA but helps control the disease. It must be taken lifelong.

This drug is also very expensive: the cost of one vial can reach 11 thousand dollars. Ksyusha, according to doctors' calculations, currently needs 19 vials of "Risdiplam" per year.

Volunteers working with the girl do not lose hope. People believe that in six months, another council can be held. If "Risdiplam" can improve the situation, doctors will eventually give permission for the long-awaited injection. Other factors must also align — in Belarus, Zolgensma is administered to children weighing up to 13.5 kg. And Ksyusha currently weighs over 12 kg.

There is also an alternative option — to seek a solution in foreign clinics. In Dubai, they are ready to administer Zolgensma if the child weighs up to 21 kg — certainly, also without guarantees. Taking the risk is possible, but such a decision must be made by Ksyusha's representative. Formally, this is her mother, but... With the foreign option, the question also arises of who will accompany Ksyusha in Dubai. There is little hope for her mother in this regard.

In any case, for now, the decision has been made to switch Ksyusha to "Risdiplam" therapy for the next six months. Part of the collected money may go towards this, but the financial question seems to be crucial. Whether Ksyusha's body will be able to recover to the level required for Zolgensma administration — is unknown.

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