The Polish Mother’s Memorial Hospital – Research Institute participates in the NFZ program for the treatment of spinal muscular atrophy (SMA). It is a severe, genetically determined disease that leads to muscle and nerve degradation. First, the hands and legs become weak, later the patient stops swallowing, breathing problems occur. Breathing support is needed. Untreated, it ends with disability and even death.
The Ministry of Health has decided to get a full refund of nusinersen – a very expensive drug that stops the progression of the disease. All patients in Poland will have access to effective treatment. It is estimated that it is about 700 people.
A five-year-old Olek was the first to be given the refunded drug. As his mother says, he was born healthy. Disturbing symptoms began to be visible in the third month of a child’s life. The child weakened, did not lift his head, stopped turning from side to side.
The disease progressed, but in 2016 the boy’s parents regained hope because nusinersen was admitted to trading in Europe. The drug stops the development of the disease, and given to patients before the onset of symptoms inhibits its development. The preparation is so expensive that few patients can afford it. Olek could get the first dose by taking part in a program dedicated to the new drug entering the market. Parents did not have to pay for the drug, but it was necessary for them to cover the costs of travel and maintenance in France and Belgium. After the onset of treatment, there was a visible improvement. But parents would have to pay for further treatment. Desperate parents considered moving to Germany, where the therapy is being refunded. Fortunately, at the beginning of the year, the Ministry of Health decided to finance nusinersen therapy.
Olek was taken under the care of specialists from the PMMH’s Department of Neurology. Its head, Dr. Łukasz Przysło, has already given the boy two doses of the preparation.
– Our patients with SMA did not have such a chance before – says prof. Maciej Banach, the President of PMMH. – We are pleased that PMMH participates in the SMA nusinersen treatment program. We invite all children to us! Do not wait with the diagnosis, if there is any suspicion of the disease, the earlier we diagnose, the more effective we will be in treatment!
More about the SMA treatment program and Olek: