The first child who received SMA treatment there thanks to state support can leave the Bethesda Hospital in Budapest on Friday, the Minister of Human Resources announced on the spot on Friday.
Miklós Kásler said: in recent years, the largest number of children with SMA were treated in "one of the strongholds of Hungarian pediatrics", the Bethesda Hospital. The treatment of this very rare, serious disease has a long tradition in Hungary. At Bethesda Children's Hospital, the life-long treatment of children with SMA with two different types of preparations began as early as 2016. Twelve children previously received the Zolgensma treatment, which is considered one of the most modern, typically through social cooperation. Three of them were from Hungary, the rest were foreigners.
Following the government's decision, starting in April, the state will provide free SMA treatment for Hungarian children for whom it is medically recommended. Two and a half-year-old Anna was one of the first to receive the treatment, and she can go home on Friday, the minister said.
The minister explained: in this case, individual fairness means that the attending physician collects all of the child's findings, and based on these, a professional committee decides whether the patient needs this treatment or another. In seven cases since April, the committee has decided that the child will receive this gene therapy free of charge; two of them have already received the treatment, and the treatment of the other five also has an appointment, he added.
György Velkey, the general director of the hospital, recalled that in October 2019 everyone got to know the name of Zente, who was the first in Hungary and in Europe to use the drug Zolgensma. After that, Levente received the treatment, followed by ten more children, for whom the price of the very expensive preparation was collected mostly through social donations. Among the foreign children, some were brought to Bethesda for treatment from the Netherlands and others from Turkey.
After a year and a half, they can already see the "very positive" effect of the drug. All twelve children are developing nicely, he said.
The director-general drew attention to the importance of early detection of the disease, which also depends on family doctors, nurses and their effective cooperation with families. After that, representatives of several professions, including geneticists and neurologists, compete with the dying neurons, he said. "Every day, every week counts," he emphasized, adding that the Hungarian child health system performed well in this race.
György Velkey thanked the entire community of the hospital, which contributes to the treatments, especially the medical director Borbála Miklós, who leads the treatment team.
At the end of the press conference, the Minister of Human Resources and the Director General visited two and a half year old Anna, who left the hospital on Friday.
Spinal Muscular Atrophy (SMA) is a rare, inherited disease caused by a defect in the SMN1 gene, which leads to the gradual loss of motor neurons in the spinal cord.
According to the press material distributed at the event, Bethesda Children's Hospital used Zolgensma therapy for the first time on October 29, 2019. Of the twelve patients treated since then, all of them have improved to a certain extent, and two little boys have already taken their first independent steps.
They recalled that Hungary is a world leader in state financing of gene therapy treatment, with gene therapy medicine becoming available on the basis of individual fairness from April.
Two-and-a-half-year-old Anna was able to receive state-supported Zolgensma at Bethesda Children's Hospital on Wednesday. In the background material, they wrote: based on their experience so far, they see a realistic chance of further improving movement skills, avoiding mechanical ventilation and preserving the ability to feed independently.
MTI
Photo: MTI/Tibor Illyés
