

Dramatic race against time to rescue Bruno's LIFE! Only the world's most expensive treatment can save our son!
Fundraiser goal: Gene therapy and accommodation in the USA, treatment and rehabilitation
Pledge 1.5% of tax to me
Pledge 1.5% of tax to me
Fundraiser goal: Gene therapy and accommodation in the USA, treatment and rehabilitation
Fundraiser description
OUR FUNDRAISER IS EXTREMELY URGENT! We have little time to collect an enormous amount of money to stop the terminal disease! Our little son suffers from Duchenne muscular dystrophy, and the only solution for him is gene therapy. We have started a dramatic race against time. Please help us save Bruno's life!
Our drama began in January 2020. After Bruno's 2-month-long battle with respiratory tract infection, we drove him to the Institute of the Polish Mother's Health Center in Lodz, where he underwent basic blood tests. Due to the poor results, the doctors decided to have Bruno thoroughly examined by different specialists. He lay on a bed for hours with an IV drip in his arm. No diagnosis was made, though. Finally, he had genetic tests done. The results came after six months.
The diagnosis was far worse than expected: Duchenne muscular dystrophy. Our world suddenly fell apart. We were beyond devastated!

Duchenne muscular dystrophy is a rare genetic disorder characterized by progressive muscle degeneration and weakness. It is caused by a mutation in the dystrophin gene (responsible for building muscle cells). As a result, muscle fibers break down. DMD affects one in every 3,300 male infants. The first symptoms include walking difficulties, followed by an inability to swallow and breathe. A wheelchair and a ventilator become essential devices for a sick child.
Most current methods are useful in treating the symptoms, not the root cause of the disease - gene mutations. Corticosteroid medicines and rehabilitation can only slow down the progress of muscle degeneration. It all sounds like a death sentence to our little boy!
This year has been groundbreaking in terms of muscular dystrophy, though! On June 22, 2023, the FDA approved through accelerated pathway the first gene therapy for pediatric patients with Duchenne muscular dystrophy (DMD) who maintained walking abilities. Gene therapy was designed to deliver into the body a gene that leads to the production of micro-dystrophin. The drug is administered as a single dose.

That day, Bruno received a chance to recover from the terminal illness! We decided to verify the conditions and possibilities of undergoing gene therapy. Since the treatment is only available in the USA, we'd have to stay there for four months. Thanks to the support of many amazing people, we found a dedicated medical facility! The team of Dr. Ghosh from the Boston Children's Hospital has qualified Bruno for gene therapy. Unfortunately, it is the most expensive drug in the world.
We have received a cost estimate of $ 5,971,000! Thanks to the Boston team's efforts, we received a discount. The price of the treatment has been decreased to $ 3,617,431 (plane tickets and accommodation are excluded), but it might rise in case any side effects or extra medical costs appear.
We cannot lose hope, though! We must try our best and start this battle despite the gigantic costs! Otherwise, we would never forgive ourselves for not taking that opportunity. My husband and I are medical rescuers - our job is to save human life. We have faced a terrible fate, and without your help, we won't be able to save our child.

This fundraiser is our last resort. On behalf of Bruno, we are asking for donations to help him win the fight for life.
-Joanna and Rafał Osłowski, Bruno's parents
