The most expensive therapy in the world to buy life for Michał, my 4 years old son!

Michał Bień
Urgent!

The most expensive therapy in the world to buy life for Michał, my 4 years old son!

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Text 0318808
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Fundraiser goal:

Treatment

Fundraiser organizer: Fundacja Siepomaga
Michał Bień, 4 years old
Słubice, lubuskie
DUCHENNE MUSCULAR DYSTROPHY
Starts on: 22 August 2023
Ends on: 01 April 2024

Fundraiser description

We are parents of two beloved twin boys, Michal and Marcel. Our sons are just 4 years-old, and they are so little, so innocent, that anyone would wish them many years of happy life. Unfortunately  Michal was diagnosed with rare genetic disorder called Duchenne muscular dystrophy (DMD). For Michal, the diagnosis was a sentence of terrible debilitating disease followed by slow but inevitably ery premature death of suffocation or circulatory problems.

That was the case just a year ago, but today there is a hope: in July 2023 FDA announced their approval for innovative gene therapy for treatment pediatric patients with diagnosed DMD, that gives them chance for life.

https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy

The therapy is approved for children 4 to 6 years of age and Michal meets all the criteria to be eligible. But the cost of the therapy is nearly 4 million USD, with 3.2 millions for the drug alone. The above means that Michal has narrow window of opportunity to undergo the treatment, and that we have to urgently collect this huge amount of money to cover the cost, as this sum is simply out of reach of us, our family and of our friends.

This is the reason why we are in desperately need of your help. We will be grateful for any donation, and if this is not possible, for passing our call for help to others who may help to save Michal's life, be it your relatives, friends, familiar businesses, fundations, coworkers and any other people of good will.

Now, when medicine and FDA decision give us the hope that we did not have before, we want to do our best to give Michal a chance for life. Michal shall grow up with his brother Marcel, they shall  support each other, and, if opportunity arise, pay this debt of good will.

We have lernt about Michal's condition about 3 years ago. At first, doctors have noticed that he is not as strong as his peers, so they started to look for the reason. Twins are often born early, and this was the case with Michal and Marcel, but this did not explain the discrepancies. Moreover, Michal wass born with skull birth defect, and we were looking to treat it. Doctors, ordered all kind of tests, including tomography scan, but already at the stage of the first blood tests it became obvious that something else is also wrong. It took some time, but finally doctors diagnosed Michal with Duchenne Muscular Dystrophy. This was really a life-changing diagnosis, suddenly our life was turned upside-down.

I've asked if it was the SMA - the only muscle-dying disease I knew at the time. "It's not SMA, but something just as cruel. Michał has Duchenne muscular dystrophy" - we heard. We started to read about the disease, about the prognosis… Nothing is the same anymore.

Naturally, as it is common in such cases, it was difficult for me to accept the truth, hoping that maybe this is just some misdiagnosis, however after talking to doctors I had to accept the reality.

Since that, I developed anxiety about his health and life. Particularly, that as soon as Michał cought a cold or any other infection he had serious breathing troubles, and I had this feeling that he is dying before my own eyes and I feel completely helpless... Simply, it was very difficult or us, both for me and for my husband, to come to terms with the new situation, with prospect of slow and inevitable death of our son.

Today the boys are four years old and you can already see the difference in their development. Michał doesn't run like his brother, needs help climbing the stairs, quickly develops fatigue, he has less strength and with each passing day he is loses it even more.

Is Michal's future, that still can be changed   if he will get the gene therapy. And this is the reason why we are begging for your help for our son. For helping Michał to live and not suffer, for helping to go to adulthood along with his twin brother. We are not hoping for miracle, wy are trying to fight for it. Simply, because we believe that there is a hope for Michał.

If you believe that you know a person, or a business, that you think might be able to help in the struggle for Michal's life, by all means, please pass this message to them too. Each and every bit of support makes a difference!

We are very thankful for your donations and any other support. Again, every bit of help is priceless as it makes the difference. Thank you for your good will and for staying with us, and for keeping Michal and our family in your prayers.

Paweł & Agata - parents of Michał and Marcel


Important information:

The cost of the drug needed for the therapy alone is $3,200,000 The estimate of  the other expenses related to the treatment and the stay in the US clinic we expect to get soon..

Donate via text

to 75365
Text 0318808
6,15 zł (including VAT)
Donate via text now

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