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Doctors prepare Ksyusha with SMA for gene-replacement therapy

A new doctors' council will be held at the end of May, the final analysis will be done abroad, according to the Ministry of Health.

The condition of three-year-old Ksenia Moiseenko, who lives in an orphanage despite having a living mother, shows significant improvement, reported Irina Zhauniaronak, the chief freelance specialist in hereditary neuromuscular diseases in children at the Ministry of Health.

Three-year-old Ksyusha Moiseenko with SMA, for whom a huge amount of money for "Zolgensma" was urgently collected by volunteers, is being prepared for gene-replacement therapy, BelTA reports.

Today Ksenia is receiving the drug "Risdiplam", which began on April 1st. "It's the end of the month now. There's very good positive dynamics, which is visible even to the naked eye," said Irina Zhauniaronak.

A repeat doctors' council will be held at the end of May. The child is currently being prepared for gene-replacement therapy. The fact is that before its start, an analysis must be taken to determine the antibody titer to the virus. The titers must be acceptable for this therapy, otherwise the manufacturer will not even ship the drug.

The analysis is performed outside the country; this is a very strict requirement of the drug manufacturer.

Ksyusha was diagnosed with type 2 spinal muscular atrophy (SMA) — a severe genetic disease that requires expensive treatment. Thanks to a quick fundraising effort, in which ordinary Belarusians, organizations, and foundations participated, almost two million dollars were raised for her treatment — an amount sufficient for the expensive injection.

But later it became known that it was not yet possible to administer it to Ksyusha. But now doctors are again giving hope.

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