"The nerves could not stand it." Why are the parents of the children with the SM

The presidential decree on the creation of the support fund for children with severe, including Orphan diseases, the Circle of Good was signed on January 6. To a large extent, it is this fund that will be assumed to provide patients with spinal muscle atrophy (SMA) with expensive drugs. But after five days, the parents of the children with SMA went to the regional Ministry of Health with pickets. “MBH Media” - about what made parents go out on the streets and how people with rare diseases are provided with therapy.

Pickets took place in Moscow, Krasnodar and Stavropol. On the posters that the parents held in their hands, there were children's names and the inscription: "Urgently need Spinraz."

“These are parents of dying children in whom the nerves could not stand. There is treatment, it is registered, and on your hands you have a baby who is getting worse every day. What would each of us do in such a situation? - explained the chairman of the board of the All -Russian Society of Orphan Diseases Ekaterina Zakharova. - All children are born with a lubricant healthy, cool, strong kids. The first signs of the disease appear after 3-6 months. But an account for children with a lumen often goes to weeks. The dead neurons cannot be returned. And there are no correct or wrong actions, from the point of view of parents. I can’t blame them for this. ”

According to her, the people who went to the pickets were not representatives of a particular organization, and the action was spontaneous. “If the fund earns and can provide drugs in a month, we do not know if our patients have a month of life. Parents hear promises, but do not see the medicine critically necessary for their child. To go to a picket is a gesture of parental despair, ”she said.

The issue of money

Spinal muscle atrophy is a hereditary neurodegenerative disease characterized by damage to the skeletal muscles and leading to increasing muscle weakness. As the disease progresses, which begins with the feet, the patient (most patients of SMA are children) consistently loses the opportunity to sit, stand, swallow and breathe. Death occurs, as a rule, due to respiratory failure. At the same time, the intelligence of patients with smell remains intact.

If you start treatment in time, you can save from the manifestations of the child's disease. But the only medicine registered in Russia that helps such children is very expensive. One spinras injection costs almost 7.9 million rubles.

“The disease develops rapidly, especially in babies with the first type of SMA. Every day, the baby is dying of new neurons, which medicine, unfortunately, is not yet able to restore, ”said Ekaterina Zakharova. - These are children whose condition worsens rapidly, every day. They have practically no time to wait. By the age of two, 80% of such children die, and for a year half is on the tracheostoma, that is, with a pipe in the throat. And they will no longer be able to turn the process. Treatment can only slow down the development of the disease. ”

In a year, patients should receive up to six injections - depending on what stage therapy is at. This makes the purchase of medicine unbearable even for wealthy parents.

In anticipation of salvation

Of the orphan diseases of the cubes, it is considered the most common. It is found in one of 6-10 thousand newborn. About 200 children with SMA are born annually in our country. In November 2020, there were 1043 patients with the SMA, 817 of them were children.

The acquisition of “spinras” should be carried out at the expense of regional budgets. Most often, parents of children with a lump are seeking the appointment of the drug through the court. Sometimes the only chance to get the medicine is to contact NPOs and declare a public collection of money.

The campaign deployed by public men and journalists in 2020 turned into an unexpected victory. In his next appeal to citizens on June 23, Vladimir Putin announced that from January 1, 2021, a progressive tax rate for persons whose income exceeds 5 million rubles a year will be introduced, and the funds received as a result of this will be aimed at treating children with orphan diseases. In November 2020, the government included Spinrase in the list of vital and most important drugs (ZhNVLP).

By November 25, the Ministry of Health has developed a draft decree “On the creation of a support fund for children with severe life-threatening and chronic diseases” and compiled a preliminary list of diseases-about 30, including the SMA-with which the fund will work.

The president announced the fact that the fund with public financing will be called a “circle of goodness” at a meeting on social issues on January 5, 2021. And the sixth signed the corresponding decree.

The founder of the children's hospice in St. Petersburg, the head of the Commission of the Public Chamber of the Russian Federation on charity and social work, Archpriest Alexander Tkachenko, headed the fund. The Board of Trustees of the Fund included actor Konstantin Khabensky, President of the Lines of Life Faina Zakharov and a member of the Board of Trustees of the Fund for Assistance to Patient Children “Be Man” Margarita Fedoseeva.

“We do not know when the fund earns how soon the applications will be considered, medicines are purchased and shipped. There are patients who need help the day before yesterday, and they all hope that the organization of purchase of drugs will not take too much time, ”Ekaterina Zakharova hopes. “All state system processes are very not fast, in general, the creation of the fund is a big step forward.”

At the request of the MBH Media, the head of the fund Alexander Tkachenko refused the situation, referring to employment.

On the way to patients

The first doses of medicine for children, according to the promises of the organization’s management, will be available in January 2021. They will be received by those who got into lists pre -collected in the regions. And since the spring, the fund will begin to accept new applications.

According to the Agency of Social Information, both parents and regional departments of healthcare or medical institutions, into which patients with lubricants managed to seek help, will be able to fill out the form for obtaining specialized support. The processing of applications will be assumed by the medical department of the fund, as well as a group of independent experts and the organization’s council.

At the same time, public organizations will continue to provide supplies and medical equipment for children with SMA. This is currently being engaged in existing specialized programs and palliative regional services, but often it is not easy to obtain lung ventilation devices.

“In order for the region to put the equipment in the right volume and the underlying quality, you often have to achieve with a lawyer. Due to the features of public procurement and the principle of import substitution, officials often acquire something that cannot be used, ”Zakharova reminded. Without an NPO or informal patient associations, to establish the necessary material support for families with children with SMA is unlikely to be possible, even if the federal fund earns, she concluded.