DMD is killing our son❗️Only the most expensive drug in the world can save him❗️

Updates

July 12, 2024, at 12:45 am

We need your support! 

If Macius doesn't receive the drug on time, the disease will take over his life! If we fail to raise money for gene therapy, his painful glance will be all we'll have left. 

I cannot even imagine that! 

My husband and I do everything to keep Maciuś in the best shape possible. We drive him to physical therapy and exercise at home. He's also taking supplements and is on a special diet. 

We were terrified to see the progress bar of our fundraiser barely getting greener. Our time was running out. When our son turns six on August 3, it will be too late for him to receive gene therapy!

And then, we saw the light at the end of the tunnel! We came across the FDA update: gene therapy can now be administered to older children and young males! 

It means that there's still a chance for recovery for our son! Also, we have more time to raise the required amount! Also, we've kept in touch with the clinic in the USA, so we'll keep you posted! 

We seek help everywhere, especially now when the opportunity presents itself to start a treatment once deemed impossible after five years of age! 

Please donate! We believe that together, we can succeed and collect this unimaginable amount of money!

It's the only way to save our son! 

Jolanta Pawlak, mom 

Fundraiser Description

A dramatic race against time has begun! Maciuś is another Polish boy who has a chance to win a battle with DMD. All we need to do is quickly raise 15 million zlotys for the gene therapy! 

Our son was born healthy, so the news about his genetic disease came as a shock to us. Although he reached developmental milestones later than other children - he had trouble lifting his head, started sitting down late - at 11 months old, and started walking at ca. 20 months old, none of us would have expected such a horrifying diagnosis. 

At first, the doctors blamed hypotonia (poor muscle tone), so we immediately started Vojta therapy. Meanwhile, Maciej also developed speech disorders and contracted frequent infections. He had trouble jumping, climbing stairs, and running. 

Being worried, we started seeing specialists. Finally, years later, we received a diagnosis that made us weak at the knees. 

Duchenne muscular dystrophy is a rare genetic disorder mainly affecting boys and is characterized by progressive muscle degeneration and results in gradual loss of physical abilities and eventually death. 

Maciuś is only five years old! We are devastated by the diagnosis, scared of the outcomes of the disease, and that we cannot do anything about it. In a few years, our son might lose all his abilities and end up in a wheelchair. 

What's worse, due to DMD, weakened respiratory muscles and cardiovascular disease will lead to breathing difficulties before Maciek turns 20 years old, which could be fatal for him. 

We want to scream: "WE WON'T ACCEPT OUR SON'S FATE!" although our words don't have that much power.

There's only one way to end this nightmare - gene therapy in the USA, which can stop or significantly slow down the progress of the DMD. However, we are running out of time to collect the money - the drug must be administered up to five years of age! If we fail, Maciuś won't receive the treatment, and DMD will continue to take its heavy toll on him. 

Besides that, our son requires constant rehabilitation and regular appointments with a cardiologist, neurologist, educator, psychologist, eye doctor, and pulmonologist. He also needs supplements and a special diet. Thankfully, following the doctor's recommendations has finally paid off. Maciek has become more fit, and his speech has improved! 

We are also parents of 7-year-old Szymon and 2-year-old Marcelinka. Our kids love one another and help Maciek as much as possible! 

Unfortunately, the reality is harsh. Medical expenses, like private doctor's appointments, are overwhelming. We cannot rely on insurance-covered visits - the waiting time is usually several years! 

We have no time to lose - we must act fast to help our child - we know that gene therapy can save his life!