Save children with SLOS!

Every day, parents of children with Smith-Lemli-Opitz syndrome (SLOS) fight to improve development, physical abilities, and provide a better future for their children.

​The stories of these families are strikingly similar. Perfect pregnancy and prenatal ultrasounds, tears of joy, and preparation for the baby's arrival. Everything changes when a child is born… Instead of its first cry, they hear a silent whimper. Tiny, flaccid bodies with fused toes have trouble sucking and require PEG feeding. Receiving a diagnosis of Smith-Lemli-Opitz syndrome (SLOS) turns their lives upside down.

“The moment you accept this situation resembles ending a grieving process after the loss of a healthy child. Realizing that your child's disability is not the end of the world.” The words of little Marysia’s mom perfectly describe what every parent of a child with SLOS feels.

​SLOS = A silent killer. What do children suffer from? 

Ordinary pain can go away after using painkillers. SLOS, however, cannot. It is an invisible and ruthless enemy. Due to a mutation in the DNA, children with SLOS are unable to produce sufficient amounts of cholesterol, an essential nutrient for proper brain development. That’s not all, though.

Abnormal cholesterol metabolism leads to the formation of toxic substances that cause irreversible damage to the nervous system, organs, and eyes. As a result, children are unable to speak or eat independently and suffer from hypersensitivity. They require constant and intense rehabilitation.

Although medicine still cannot offer an effective treatment for SLOS, there is a light at the end of the tunnel - gene therapy. Support from good-hearted people is needed, though! It’s an all-hands-on-deck situation! 

The disease is rare, so for most pharmaceutical companies, developing a gene therapy for so few children is not worth the effort. Their parents don’t give up, though, searching for possible solutions everywhere they can. They managed to reach Leszek Lisowski, a Professor and a world-renowned scientist. He has been working on developing gene therapies for children with rare disorders. His Gene2Cure Foundation’s objective is to create 10 gene therapies for 10 incurable diseases, including SLOS. 

The treatment is about creating a special vector that delivers a correct copy of the gene directly to the damaged brain cells. As a result, the repaired DNA launches the production of cholesterol! 

The research has already started. During the laboratory testing phase, the scientists successfully generated at least one vector prototype, which has been approved for clinical trials. It means that life-changing gene therapy might soon be developed! It’s the last resort for children with SLOS, and the first step to treat incurable diseases! 

The only obstacle is money. A lot is needed to finish the project. It comprises preclinical studies, the production phase, and finally, delivering therapy to the patients. The total required amount is 5 million euros. While a single family cannot afford it, a contribution made by thousands of good-hearted people can make a miracle happen and save children with SLOS! 

We have started a fundraiser to offer children a chance for a better life, which they deserve. We need your help, though. Developing gene therapy is a complex and expensive process, but we need to fight for it! For the sake of all children who need help.​

Join our fundraiser and change the lives of children suffering from SLOS! Give hope!

Meet our Beneficiaries with SLOS!

💚 Jaśminka (opens a new tab)

💚 Klara (opens a new tab)

💚 Kubuś (opens a new tab)

💚 Nikodem (opens a new tab)

💚 Ola (opens a new tab)

💚 Wiktoria (opens a new tab)