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State Refuses Funding for Treatment of Child with SMA, Donations Now Being Collected

Júlia Tar 2022.01.11.

Because of the regulations in Hungary, a boy with SMA did not get state aid to fund his recovery. Now his parents are collecting donations.

Last May, a little girl who was diagnosed with spinal muscular atrophy (commonly called SMA) was the first to receive aid from the Hungarian state for her treatment. The medicine Lili received is called Zolgensma and costs HUF 700 million (almost EUR 2,000,000). Since then, several other children received the aid. Unfortunately, this is not the case with a little boy who has experienced not only the beneficial effects but also the side effects of another medicine.

Fact

Spinal muscular atrophy (SMA) is a genetic condition that causes muscle weakness and atrophy (when muscles get smaller). In SMA, the nerves that control muscle strength and movement break down. These nerves (called motor neurons) are in the spinal cord and lower part of the brain. They can’t send signals from the brain to the muscles to make them move. Because the muscles don’t move, they become smaller (atrophy).

SMA can affect a child’s ability to crawl, walk, sit up, and control head movements. Severe SMA can damage the muscles used for breathing and swallowing.

For possible signs, symptoms, and other information, click here. For the SMA Hungary Foundation’s website, click here.

Tomika received another medicine called Spinranza. As his parents wrote in a Facebook post, “Sparing no time and energy, we searched both at home and abroad for various aids and rehabilitation options to complement the Spinranza treatment and ensure Tomika’s development in the most efficient way possible. In the beginning, we traveled thousands of kilometers for Spinranza itself, as no treatment was available in Hungary at that time. Tomika received the first 4 doses in Belgium.”

State Funds SMA-Afflicted Child's Treatment for First Time
State Funds SMA-Afflicted Child's Treatment for First Time

No longer will Lilike’s parents need to ask for donations for the 700-million-forint (EUR 2 million) treatment.Continue reading

They say that while Spinranza helps a lot, “patients with SMA type 1 – who also have swallowing problems – may experience a deterioration in their ability to swallow.” This happened to Tomika as well. “This is how Zolgensma came into the picture, as the last chance for real progress and improvement, as it has been proven to help more than the previous treatment,” his parents write.

The National Health Insurance Fund Management “set the aid line at 13.5 kg and 3 years of age for this medicine,” Tomika’s parents wrote. However, “last year Zolgegsma received the European licenses and a registration certificate. In the pedigree and in the guide it is not the age that is mentioned, but the body weight. The manufacturer of the drug has calculated the dosage amounts up to 21 kg. This is the basis on which some countries (e.g. Italy, Germany) have started to treat older children up to 21 kg, regardless of age.” Despite the treatments in other countries, according to his parents, the Hungarian regulations do not allow the treatment in Tomika’s case since he is 16 kg.

Six More Children With SMA to Receive Free Gene Therapy
Six More Children With SMA to Receive Free Gene Therapy

"We have become world leaders in public funding for gene therapy treatment," the National Health Insurance Fund Management said.Continue reading

This is why his parents see donations as the only option. Those who would like to help can do so in many ways.

Featured image: Tomika celebrating his 5th birthday. Photo via the Facebook page called “Together for Tomika – SMA1 – Foundation for the Recovery of Tamás Dominik Galó”