The Polish Mother’s Memorial Hospital – Research Institute participates in the NFZ-funded program for spinal muscular atrophy (SMA). This is a severe, genetically determined disease that leads to muscle and nerve degradation. First, the arms and legs weaken, then the patient stops swallowing, breathing problems start. Breathing support is necessary. Untreated, it ends with disability and even death.
The Ministry of Health decided to fully refund Nusinersen – a very expensive drug that stops the disease from progressing. All patients in Poland (estimated 700 people) are to have access to this effective treatment.
The beneficiaries of the program are, among others, Michał and Tosia who are siblings. He drug had been administered to them before the disease developed. Effects? After the fourth dosage they passed HFMSE functional tests with flying colours, which indicates that their physical fitness improves.