Parents of children with Duchanne's muscular dystrophy were looking for rescue in the Sejm

There are about 400 children in Poland who suffer from Duchnne's muscular dystrophy. A new American drug has recently appeared, which finally gives some hope, but therapy costs several million zlotys. Internet collections do miracles, but they don't always succeed. This week, parents were looking for their children in the Sejm.

Ignas and his parents came to the Sejm this week. Liss and other parents wanted their politicians to listen to them. – We are alone with the disease of our children – said Mr. Bartłomiej Liss, dad of a sick boy … – The Polish state is fighting for every child to live, but if this child survives, he is born, parents are left to the fate – said Jolanta Pawlak, mother of sick Maciek.

Children like Ignas are around 400 in Poland. We showed their history in our materials. Muscle dystrophy Duchanne is a rare genetic disease that only affects boys and leads to irreversible muscle atrophy.

– Slow agony. This is the decline of this disease. Man is waiting for a child and then gets such a diagnosis and cannot look at the future of his child with hope – says Alicja Orlik, mother of the sick Adam.

Hope gene therapy

New gene therapy developed in the United States gives hope to the sick. – It is already registered for boys over the age of four. It would slow down the development of the disease. These are the initial test results – indicates prof. Magdalena Chrościńska-Krawczyk, head of the Clinic of Pediatric Neurology of the University Children's Hospital in Lublin.

The results are preliminary, there is not many data, but it is a lot for parents. – We believe, we believe, because it just keeps us in strength – emphasizes Mr. Bartłomiej Liss, Dad Ignacy.

That is why they have to become millionaires for their children, because millions cost the therapy they assume. On the internet, these are record -breaking for amounts that it is difficult to even imagine, let alone accumulate.

– We collected three million zlotys during the year. In fact, we are already finished with this collection – confesses Jolanta Pawlak. – 16 million per child. We also answer such questions in social media. Is it worth it? – says Alicja Orlik.

American therapy is not reimbursed in Poland. The preparation has not been allowed to the EU market. – This is great hope. I understand the social side and parents of these children that they do not have to fully understand the issues of registration of the drug, clinical trials – admits Agnieszka Gorgoń -Komor, MP of the Civic Coalition.

There is a lot to be done. Parents came to the Sejm with their ideas for financing and postulates for rehabilitating. Politicians now announce meetings with the group of relevant people, decision -makers and experts. – We have to deal with therapy as soon as possible, because time, unfortunately, plays our disadvantage – emphasizes the father of the sick Ignacy.