Since the beginning of 2020, three children with SMA in Russia have died without waiting for treatment

In Russia, since the beginning of 2020, three children with spinal muscular atrophy (SMA) have died without receiving the necessary treatment.

Yulia Korsunskaya, an employee of the hotline for drug provision of the SMA Families Foundation, told Takim Dela about this. There are three drugs in the world that can slow down or stop the development of certain types of SMA - Spinraza, Risdiplam and Zolgensma. Spinraza is registered in Russia; regional authorities must pay for the multi-million dollar treatment.

Activist from Krasnoyarsk Nadezhda Bolsunovskaya reported on Facebook that a boy with SMA who did not wait for Spinraza died in the Krasnoyarsk Territory. “What is happening now with the treatment of children with SMA in the region is hell,” Bolsunovskaya wrote. According to another Krasnoyarsk activist, Olga Fuks, this boy’s family has another child with type 2 SMA, and he also requires treatment. The SMA Families Foundation told Takim Dela that a total of 13 people with SMA live in the Krasnoyarsk Territory.

The mother of the deceased boy, Svetlana Rukosueva, in an interview with MK Krasnoyarsk, said that the drug Spinraza, registered in Russia back in August 2019, is still not available in the Krasnoyarsk Territory. At the hospital, when asked when her child would be given the medicine, they answered: “Don’t wait, it won’t be available,” Rukosueva claims. Roszdravnadzor told Rukosueva that Spinraza was in stock.

“Such cases” sent inquiries to the Ministry of Health of the Krasnoyarsk Territory and the regional prosecutor’s office. No responses had been received at the time of publication.

Spinal muscular atrophy is an incurable genetic disease that causes damage to the nervous system and gradual muscle atrophy. The first year of treatment with Spinraza costs 45 million rubles, then treatment will cost 20 million rubles per year. Such therapy is necessary until the end of the patient's life.

The 2020 federal budget does not include funds for the treatment of patients with SMA—the responsibility to purchase Spinraza lies with the regions. Director of the SMA Families Foundation Olga Germanenko told Takim Dela that this winter the regions began providing therapy to patients for the first time, but so far we are only talking about single purchases - hundreds of other patients are left without the necessary drugs.

The director of the portal “Takie Dela” and the charity foundation “Need Help” Mitya Aleshkovsky appealed to the Prime Minister of the Russian Federation Mikhail Mishustin with a request to allocate the necessary funds for medicines for patients with SMA. Mishustin plans to reform the system of social support for the population, transferring it to a targeted model. According to Aleshkovsky, families with children with SMA are ideal for targeted assistance.

In January, the State Duma held its first working meeting on a bill to help people with SMA. The document proposes to amend the Federal Law “On the fundamentals of protecting the health of citizens of the Russian Federation” so that patients with SMA can receive therapy at the expense of the federal budget. Also, the SMA Families Foundation asked Russian President Vladimir Putin to include SMA in the program of high-cost nosologies.