Gene therapy in the fight against SMA and rehabilitation
Ends at: 30 October 2021
The diagnosis ruined our family life. We need the highly expensive therapy, which will hold the dreadful disease back. There is a chance for Maja, but we need thousands of good hearts to reach it.
When she came into the world, she became indispensable to our family. In our quiet prayers, we've always asked only for health for us and our children. Maja grew up just like her peers to a certain point. Her health assessments took place rarely due to the pandemic but all in all they didn't reveal any anomalies. Unfortunately, the longer gap between vaccinations and no usual access to healthcare coincided with disturbing symptoms.
As a mother I had an intuition that something must be wrong. At that point, I was told to wait and not to exaggerate that much. I saw that Maja sits only when she was sat down; she doesn't keep her back straight, she doesn't crawl and she doesn't even catch her legs just as other children. The first consultations with physiotherapists confirmed my own concerns. Though, at that time they took a low muscle tension into consideration, as well. While the tests were keeping up, I was waiting tensely for more information.
I remember when I heard these words for the first time: SMA. I froze. When I received the test results on 17th May, I literally was dumbfounded. I was even entirely sure that it had to be a huge misunderstanding. But yet, we faced the harsh reality.
Now we have to make our first difficult decisions- the choice of physical therapists, the purchase of equipment, the further treatment and medical consultations.
Everything is changing at an alarming rate! We are trembling with fear thinking about Maja’s future. We know one thing: she needs a gene therapy to provoke her body to produce a protein, which now it doesn't produce because of the genetic defect. That protein is fundamental to right functioning of the muscles.
The total cost of that therapy is head-spinning. The awareness that every day is crucial for fighting against the disease is extremely difficult for us. That's why, we ask for help, which can give our daughter the chance for psychomotor aptitude and normal life. We want to accompany her while taking her first steps instead of buying a wheelchair. We want her breathing to be stable and her meals not to be served through a feeding tube.
We cannot be alone during this battle - we have to gain the support of thousands of angels with whom we will go for Maja’s future. You can turn out our daughter’s fate, we really count on You.
When Maja looks at me with trust I want to scream out our powerlessness, pain and terror… Although, we have only found out about the disease, we receive new information about things well need to face day by day. Our fear thrives... If we don't deal with the disease, everything we hoped for will be gone.
I'm so dreadfully scared that I will have to control every kilogram of her weight to make sure that the gene therapy is still possible, as the treatment can take place only when a child is under 13,5 kilograms. I'm aware that there is a long and a tough road ahead but I know that thanks to your help everything is possible!
This diagnosis doesn't have to be a sentence. It's all or nothing for us! That's the reason why we fight for everything the best for our daughter. Thanks to you we can make our dream come true and achieve the impossible, make Maja’s life not to be doomed to misery, pain and restrictions. Your good heart can change a lot!
Maja’s parents, Natalia and Dawid
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