We are raising money for our son Alex who has a spinal muscular atrophy type 1. He is only able to lie, but with a new gene therapy Zolgensma he could be able to sit, crawl and maybe even walk! Please help us get it!
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 Alex stopped lifting his legs to his stomach at the end of the fifth month. The real condition of our baby 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.
At Boston Hospital, they identified Alex as suitable for Zolgensma. The price of Zolgensma itself is $ 2.185 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.
Monika, Marek, Diana & Alex Brdarsky
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