Cure for Alex.Alex was born on July 20, 2018. For the first months we had a great time with him and his little sister. Gradually, however, we became worried that he can not turn himself into a belly. The beautiful season and the enjoyment of a little baby ended. A huge blow came when Alexstopped lifting his legs to his stomach at the end of the fifth month. The real condition of ourbaby was much worse than we thought. Spinal muscular atrophy (SMA) grade 1. A diagnosis that has frustrated all our ideas of normal life. SMA leads to the gradual loss of muscle cells (and thus the ability to move) and subsequent death within two years of age. Affected are lower limbs, difficulty with swallowing and later with breathing. Patients with SMA either do not acquire the ability to sit, stand and walk at all or, if so, gradually lose this ability. In May 2019, the US Food and Drug Administration (FDA) approved Zolgensma. It is the new and only gene therapy for children with SMA under the age of two, available so far only in America. In Slovakia, only “supportive” treatment with Spinraza, which has a different mechanism of functioning than Zolgensma, is available. Spinraza does not cure SMA, but slows down or stops the progression, is the deterioration of health. Zolgensma focuses on the genetic cause of SMA. It replaces the function of the missing SMN1 gene with a new SMN gene, in which motoneuron cells that have not died can survive, function and be maintained.
Alex could achieve a significantly higher quality of life after receiving this medicine.At Boston Hospital, they identified Alex as suitable for Zolgensma. The price of Zolgensma itself is $ 2.1 million. With this single dose price, Zolgensma is the most expensive drug in the world. But we are not giving up and we have a challenging journey to get Alex’s cure for his illness. But it’s important that Alex gets the medicine until his second year, that is, by July 2020. Although we know that time is passing fast, having good people around us moves us forward and we believe that if we join forces, everything will be possible! If we do it together, Alex will start a new life in a new way – steps. And one day we’ll tell him that the whole world saved him from a genetic failure that could break his life.
Thank you for your help, support, and for every sharing of Alex’s story.
Alex’s parents Monika and Marek Brdárskyodičia