Six-month-old Hansel from Chrzanów (Lesser Poland Voivodeship) suffers from spinal muscular atrophy. Since June, his relatives have been collecting money for a gene therapy that is very expensive and unavailable in Poland, which may save his life. Until Saturday, over nine million zlotys were collected. The fundraiser was supported by over 178,000 people.
Jan was diagnosed with SMA, i.e. spinal muscular atrophy. The disease progresses very quickly and is aggressive. The boy has problems with swallowing and breathing, and requires the help of a respirator. There is one reimbursed therapy for Johnny in Poland. A drug is injected into the boy’s spine every few months. However, it is associated with great pain.
Gene therapy can help a child, but it is very expensive and not reimbursed by the National Health Fund. It is a one-time infusion of the preparation, which costs over PLN nine million. The medicine should be given before the child weighs 13.5 kg. That is why Jan’s parents launched a fundraiser online. It has been run on the siepomaga.pl website since June. Until Saturday, over 9 million 427 thousand zlotys were collected.
What is the therapy? – It’s about supplying the body with a gene fragment that is missing in children with spinal muscular atrophy. Giving it well in advance allows these children to function practically properly – said in “Fakty TVN” Dr. hab. Magdalena Chrościńska-Krawczyk from the Department of Children’s Neurology of the Children’s University Hospital in Lublin.
Jaś is not the only child in Poland suffering from SMA. Annually, about 50 children suffering from this disease are born. The Ministry of Health announced price negotiations with the drug manufacturer. Ultimately, the decision whether gene therapy will be reimbursed rests with the ministry.
Main photo source: TVN24