According to Novaya Gazeta, the Circle of Kindness charity foundation, created to support children with rare diseases, will pay for the treatment of six babies, including the hero of Novaya Gazeta's publications, Martin Wulpe.
“Martin has been approved for having Zolgensma at the expense of the Circle of Kindness fund! Today we cry, but we cry with happiness. We have made our path to these words for almost during five months”, - the newspaper quotes the message of the boy's mother, Margarita Wulpe.
Initially, treatment with Zolgensma was approved for only two children. The experts recommended to the rest of those in need, including Martin Wolpe, to purchase other medicines - "Spinraza" or "Evrisdi" (risdiplam). Unlike Zolgensma, which heals a child from SMA after a single injection, other drugs, which are also very expensive, must be taken regularly and for life.
After the experts made their decision at the end of June, representatives of patient organizations, parents and journalists asked the foundation to return to the issue again. And after that, at an extraordinary meeting on August 2, the Expert Council approved the purchase of Zolgensma for four more children.
The cost of one dose of Zolgensma is more than $ 2.1 million (about 160 million rubles). The most expensive drug in the world, developed by Novartis for the treatment of SMA, has not yet been registered in Russia. Its use is effective for the treatment of children under two years of age or weighing no more than 21 kg. Further, its use is considered ineffective.
Due to the lack of registration of the drug in the Russian Federation, earlier parents of sick children were forced to apply to charitable foundations, trying to collect "cosmic" sums to buy the necessary drugs. At the beginning of this year, on the initiative of the President of the country, the Circle of Kindness Foundation was created to solve this problem. However, the first applications were admitted to consideration only after seven months.
Many families have to sue the state to get the treatment they need. And decisions are not always made in favor of patients. So, in June of this year, the Second Cassation Court upheld the decision of the Tverskoy District Court of Moscow, which previously refused to provide a two-year-old girl with spinal muscular atrophy (SMA) with the drug "Spinraza" at the expense of the regional budget.
The family went to court, because the officials of the health department did not give guarantees for the provision of the child with the drug from 2020. However, the department, says the girl's father Yakov Redko, provided Spinraza without delay.
Family lawyer Boris Malakhov said that legally the family should not depend on the will of the department. The Spinraza injection should be given every four months. The child received the last injection of the drug on April 26, the next one is scheduled for August, but there are no guarantees for this. The girl needs "Spinraza" throughout her life until she is canceled for medical reasons.
Meanwhile, in Moscow, prosecutors began testing after the death of a five-year-old boy with SMA who did not receive the drug. The drug was prescribed to him in 2019, but he never received it.
Earlier, parents of children in Tatarstan faced the same problems. A nine-year-old girl from the city of Arsk was prescribed this drug back in 2019, but so far she has not received it. The child's parents appealed to the European Court of Human Rights, which ordered the Russian authorities to buy medicine for the child.
Kostya Voronikhin from Achinsk, Krasnoyarsk Territory, died on March 9, the day before his first birthday: on March 10 he would have turned a year. A month ago, he was assigned Spinraza for health reasons. But he was not treated. In the Krasnoyarsk Territory, this is the second death of a child with SMA who was deprived of treatment. On January 30, Zakhar Rukosuev died in the intensive care unit of the Krasnoyarsk Regional Children's Hospital. He was two years and eight months old. At one year and four months, he was diagnosed with SMA - spinal muscular atrophy. Death occurred as a result of cardiac arrest. There are more and more such stories.
In February, in Krasnoyarsk, police officers drew up a report on the father of a two-year-old child who had died from SMA. He went out to the building of the Krasnoyarsk Territory administration with a picket. The poster he was holding read: "Governor, who will be responsible for the death of my son?" Nearby was a wheelchair with a portrait of a deceased boy.
While officials are delaying the resolution of the problem, the list of victims of spinal muscular atrophy is growing. And those who are sick understand that the same thing can await them in the near future, if the necessary medicines are not available.