gene therapy to fight SMA1, rehabilitation
Ends on: 05 October 2022
Urgent! We are fighting for the life and health of our little son! There are two dangerous opponents against us - SMA1 and time! We will not win without you, and the disease will consume Leon’s life! The disease has crept into our lives quickly and imperceptibly - if we want to fight it, we must repel this attack and pray for a miracle!
It is a terrible feeling when you are a parent and you know that the smallest and weakest person in the family is in mortal danger and you cannot do anything to help him.
We cannot helplessly watch SMA drain our breath and destroy our muscles. We have one chance, so we are asking you for help!
Throughout my pregnancy, I felt well, I was glad I would deliver soon. We already have an older son, who watched with interest my growing belly and waited for his brother. Finally, the culmination of pregnancy - long awaited delivery and great happiness! Initially, everything was great. Our son was born on April Fool’s Day, April 1st - big, strong and healthy. We came home absolutely happy. Nobody expected that our pure happiness would disappear in a blink of an eye.
Anxiety turned to fear ...
For the first month, nothing frightened us! We took care of Leonek just as we did with our older son. We kept telling ourselves that Leoś was lazy, that he might start to be more mobile, that he needed more time ... We felt deep inside that something bad was going on, but we didn't know that our son was so sick! I remember the day when I decided to watch him more closely, when I followed his every move, hoping that maybe I was imagining things.
Time passed and the anxiety grew - we found a pediatrician who referred us to the hospital. On June 2, our family's life turned upside down. In the hospital corridor, we learned that our two-month-old son Leon suffers from a genetic disease - SMA type 1, the worst form of spinal muscular atrophy that causes his muscles to die every day. This disease has nightmarish faces. It takes away the strength of the muscles, breath and the possibility of swallowing. If the disease is not faced, the child is doomed to a respirator or to death!
On June 4, Leon received the first dose of the reimbursed drug. This medicine stops the progression of the disease, but you must take it every four months for the rest of your life. Each infection of our son can weaken his muscles, which in turn will lead to problems with swallowing or breathing.
SMA1 is taking his life and we cannot wait
The diagnosis will echo in our heads forever, but we can't sit down and cry. We must fight until it is too late!
In Poland, our son can receive a drug that only slows down the progress of the disease. Leon has to take it straight into the spinal fluid for the rest of his life. However, there is a chance to save our baby. There is a drug that, once administered intravenously, will make Leon develop normally. Our son has a great chance because the disease was detected early. No treatment can undo the havoc of SMA, but if we give the drug now, Leon can function very well!
Unfortunately, the drug is absolutely financially out of reach for us. It is the most expensive medicine in the world, costing PLN 9.5 million! The drug, which is a virus, causes the body to re-produce the protein necessary for muscle development. Unfortunately, the drug can only be given up to a certain body weight - it is 13.5 kg. We are starting to fight the worst - that is, time that is inexorably quickly running out.
Without you, we are doomed to failure, but if you stay with us, we will be able to save Leon's health! His life can be changed, but you have to hurry! If you help us, if we make the fundraiser available and mobilize all our strength, we will surely succeed!
We are begging you for help, because the time is running out. Thank you for all the help and the support. Good always comes back, and it comes back stronger!
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