Two more children with SMA from Russia benefited from treatment with Zolgensma

Two children from Russia with spinal muscular atrophy (SMA) won free treatment with Zolgensma. This was announced by the head of the SMA Families Foundation, Olga Germanenko.

“One of the lucky ones is Polina E. from Bashkiria, and the second family is not present on social networks and does not plan to reveal who they are and where they are from (they do not collect [for the medicine]),” Olga wrote.

At the end of 2019, the Swiss pharmaceutical company Novartis AG launched a free distribution program for the drug Zolgensma. It is valid in countries where sales of the drug have not been approved. Once every two weeks, an independent commission draws lots among applications submitted by doctors. The first child from Russia benefited from treatment in this way at the end of July.

In April, two children with SMA received Zolgensma for the first time in Russia. There is only one drug registered in the country for the treatment of SMA - Spinraza; therapy must be continued throughout life. Its purchase must be paid for by regional authorities, but due to the high cost of the drug, not all patients receive treatment . For treatment with Zolgensma gene therapy, one injection is sufficient. This is the most expensive medicine in the world. The cost of the injection is estimated at $2.1 million to $2.5 million.

Read more about Zolgensma and other drugs used in the treatment of SMA in the TD article .