PUS3 Foundation

17/05/2026

There has NEVER been a conference like this before. 🔥

For the first time, an emerging therapy for children with PUS3 syndrome will be featured as a topic at an international conference dedicated to gene therapies. ❤️

Our partner, the Gene2Cure Foundation, is organizing a unique event that will bring together scientists, physicians, and families affected by ultra-rare genetic diseases.

Among the speakers will be Professor Leszek Lisowski, PhD, MBA, who is actively involved in developing a gene therapy for children affected by PUS3 syndrome. During the conference, he will present: “How gene therapy works: From vector engineering to clinical delivery.”

We are also honored that Dr. Špela Miroševič, PhD will speak at the conference with her presentation: “Why Gene2Cure exists: Building beyond a single therapy.”

Another featured speaker will be Asst. Prof. Duško Lainšček, PhD, who will present: “PUS3 and GAND syndrome mouse model development and preparing for efficacy studies.”

Just a few years ago, it was hard to imagine that PUS3 syndrome would even enter the world of advanced genetic research. Today, a therapy is actively being developed.

Thank you to everyone who continues to walk this journey with us. ❤️

More information 👉 https://gene2cure.com/gene-therapy-conference-2026/

27/03/2026

PUS3 mutation featured in Fakty TVN 🎥 bringing wider attention to this ultra-rare genetic condition.

👉 https://www.facebook.com/reel/908904491935950

When large pharmaceutical companies deemed this condition “not commercially viable,” Polish scientists stepped in to take action — working on developing a dedicated gene therapy.

This is a true race against time, with an estimated cost of nearly €5 million.

The PUS3 Foundation, established by parents of affected children, is actively raising funds to support this groundbreaking research.

We encourage you to support this initiative and help accelerate the development of a life-changing therapy.

👉 https://www.pus3foundation.org/donate

19/03/2026

PUS3 Syndrome in the Media – Report on TVN24 🎥

On March 18, a report about children with PUS3 syndrome—an ultra-rare genetic condition—was broadcast on TVN24 👉 https://tvn24.pl/zdrowie/tylko-12-osob-na-swiecie-choruje-tak-jak-one-polscy-rodzice-walcza-o-terapie-genowa-dla-swoich-dzieci-st8958481

The material highlights the daily reality of affected families and emphasizes the urgent need to develop gene therapy. Featured speakers included
Prof. Leszek Lisowski,
Prof. Robert Śmigiel,
and Jan Kieszczynski, President of the PUS3 Foundation.

The PUS3 Foundation is currently leading a project to develop a gene therapy for children with PUS3 syndrome, as no effective treatment exists today.

We encourage you to support this initiative:
👉 https://www.pus3foundation.org/donate

Dwanaście osób na świecie, połowa w Polsce. Gdy system uznaje ich chorobę za "nieopłacalną", rodzice i polscy naukowcy biorą sprawy w swoje ręce i tworzą własną terapię genową. To wyścig z czasem wart 5 milionów euro - o przyszłość sześciu polskich rodzin. Fundacja PUS3 Foundatio...

28/02/2026

February 28th is International Rare Disease Day. At PUS3 Foundation, we believe that being rare does not mean being insignificant.

Every voice deserves to be heard, and every story deserves to be understood. Today, we are shining a light on those who face challenges every day that the world too often forgets. 💡

Let this day serve as a reminder that being rare is not about isolation—it is about a unique strength that connects us all. 🤝


💖 💚 🩵 💜

27/02/2026

PUS3: Fixing a bug in the DNA code. Polish scientists are developing a breakthrough drug for an ultra-rare disease of Polish origin. 👉Link to the article on LinkedIn in the comment.

27/02/2026

🎥 EPISODE 2
Gene therapy for PUS3 syndrome?
Why does a world-class scientist decide to create it?

22/02/2026

🎥 Episode 1
Who knows how to create a cure for PUS3?
Prof. Leszek Lisowski.

24/01/2026

Low muscle tone is just one of many PUS3 Syndrome challenges— but gene therapy can target the root cause.

Help us bring it to children faster.

Donate here 👉 https://www.pus3foundation.org/donate

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Obniżone napięcie mięśniowe to tylko jedno z wielu wyzwań Syndromu PUS3. Terapia genowa dociera do źródła problemu.

Wesprzyj nas i pomóż szybciej udostępnić ją dzieciom.

Przekaż darowiznę 👉 https://www.pus3foundation.org/pl/donate

28/12/2025

Our official fundraising campaign is now live 💚
👉 https://www.pus3foundation.org/donate

Thank you to everyone with a big heart who supports our mission— by donating, sharing, and standing with us 💚

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Nasza oficjalna zbiórka właśnie wystartowała 💚
👉 https://www.pus3foundation.org/pl/donate

Dziękujemy wszystkim ludziom o wielkich sercach za wsparcie — darowizny, udostępnienia i bycie z nami 💚

22/12/2025

Meet Victoria — one of Our Heroes ✨

Victoria may not walk or speak yet, but she connects with the world in her own truly beautiful ways 💚

She loves the swimming pool 💦, is fascinated by mirrors ✨, and lights up at the sound of birds singing 🐦

You can follow Victoria’s adventures on social media — on Facebook and Instagram 📸

Because every hero has a story, and Victoria’s is full of wonder 💫



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Poznajcie Victorię — jedną z Naszych Bohaterek ✨

Victoria nie chodzi ani jeszcze nie mówi, ale w swój własny, piękny sposób reaguje na świat 💛
Uwielbia basen 💦, fascynują ją lustra ✨ i rozświetla się na dźwięk śpiewu ptaków 🐦

Jej przygody możecie śledzić w mediach społecznościowych — na Facebooku i Instagramie 📸

Bo każdy bohater ma swoją historię, a historia Victorii jest pełna małych cudów 💫

17/12/2025

PUS3 is an ultra-rare neurodevelopmental disorder caused by mutations in the PUS3 gene 🧬

For affected children and families, it means lifelong challenges and unmet medical needs.

Learn more, help raise awareness, and support our mission 💚
https://www.pus3foundation.org/

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PUS3 to ultra-rzadka choroba neurorozwojowa spowodowana mutacjami w genie PUS3 🧬

Dla dotkniętych nią dzieci i ich rodzin oznacza to wyzwania na całe życie i brak wsparcia medycznego.

Dowiedz się więcej, pomóż zwiększyć świadomość i wspieraj naszą misję 💚
https://www.pus3foundation.org/