Zachráňme Vilyho | Let's save Vily

Ako sa Vilkove ochorenie prejavuje?

Vily má veľmi vzácnu formu Duchennovej svalovej dystrofie. Je to genetické ochorenie, pri ktorom jeho telo nedokáže vytvárať dystrofín – bielkovinu, ktorá drží svaly pevné a zdravé. Keď chýba, svaly sa postupne ničia a slabnú. Najskôr sa to prejaví na svaloch nôh, panvy a ramien. Deti postupne strácajú silu pri chôdzi, behu či vstávaní. Neskôr ochorenie zasiahne aj ruky, dýchanie a dokonca aj srdce. Bez liečby deti okolo ôsmeho až dvanásteho roku života väčšinou prestanú samostatne chodiť a postupne prichádzajú ďalšie vážne komplikácie. Toto ochorenie dnes, žiaľ, vedie k zlyhaniu dýchacích svalov či srdca a tieto deti nakoniec zomierajú. Z toho dôvodu musíme konať čo najrýchlejšie.

Je možné zachrániť Vilyho?

Hneď ako jeho rodičia zistili, čo mu je, Vilkov otec našiel a oslovil medzinárodný tím vedcov a lekárov, ktorí vedia pripraviť špeciálnu liečbu. Pracuje na nej tím svetových kapacít – profesor Wein z Francúzska, špecialista na génové terapie; profesor Škorvánek z Lekárskej fakulty UPJŠ v Košiciach; a docentka Šoltýsová z Univerzity Komenského v Bratislave. Títo ľudia a ich spolupracovníci patria medzi svetových odborníkov v ich oblastiach a vďaka ich znalostiam a skúsenostiam, sa náš Vily môže uzdraviť. Liek, ktorý vyvinieme pre Vilka, môže pomôcť aj iným deťom na svete s podobnou mutáciou. Naša snaha tak nemusí zachrániť len jedného chlapčeka, ale otvoriť dvere celému výskumu.

Čas hrá proti Vilkovi

Zlomové sú nanešťastie prvé štyri roky života detských pacientov, Vilko má, žiaľ, už dva. Každý deň mu ubúdajú svaly, ktoré sa už neobnovia. Čoraz ťažšie chodí, padá a zo dňa na deň horšie vstáva. Aby sme ho zachránili, liek by mu mal byť podaný čo najskôr.

Podporte vývoj lieku a záchranu Vilyho

Celková suma potrebná na vývoj lieku vďaka spolupráci s vedcami a záchranu života Vilyho je 3 963 500 eur.

Prvý míľnik: 880 000 eur   
Výroba prvej dávky liečiva v špecializovanom zariadení v USA a overenie jeho účinnosti na Vilkových bunkách a modeloch, zrealizované odborníkmi z inštitútu génových terapií v Nantes – za spoluúčasti slovenských vedcov.

Druhý míľnik: ďalších 1 012 886 eur

Realizácia štúdie bezpečnosti a toxicity liečiva v špecializovanom GLP laboratóriu, spolu s výrobou a validovaním liečiva pre podanie, ako aj príprava zodpovedajúcej dokumentácie pre schválenie realizácie klinickej štúdie na Slovensku.

Tretí míľnik: ďalších 1 800 000 eur

Výroba finálnej dávky, takzvaného cGMP grade liečiva v špecializovanom zariadení v USA, potrebnej pre podanie liečby Vilkovi, spolu s prípravou nevyhnutnej dokumentácie a certifikáciou pre použitie v klinickej štúdii

Štvrtý míľnik: ďalších 270 614 eur

Náklady spojené s realizáciou klinického výskumu, a teda s podaním liečiva a následnou zdravotnou starostlivosťou o Vilka – realizované v Univerzitnej Nemocnici L. Pasteura v Košiciach v spolupráci s Lekárskou fakultou UPJŠ v Košiciach a profesorom Weinom s jeho tímom.

Každý príspevok nás posúva bližšie k liečbe, ktorá môže Vilymu zachrániť život. Ak nám ľudia pomôžu, máme šancu zastaviť túto zákernú chorobu.

Ďakujeme vám, že nám pomáhate zachrániť Vilyho!

Vilko lives with Duchenne muscular dystrophy

Vilko is only two years old, yet he’s already a little boy who captures the hearts of everyone around him. Even though he lives with Duchenne muscular dystrophy, his days are filled with laughter, curiosity, and pure joy for life. He’s a happy child who, with a sparkle in his eyes, builds Lego creations as if he were shaping his own colorful world brick by brick. He flips through his picture books with excitement, discovering the world around him with a child’s endless curiosity.

The most beautiful moments he shares with his family—especially with his older brother, with whom he laughs, plays, and enjoys quiet moments of closeness. He loves helping in the kitchen because he wants to be part of everything that happens around him. Wherever he goes, Vilko brings joy and calm. He loves his parents, his brother, and animals with all his heart. He’s living proof that true strength isn’t measured by muscles, but by the heart—and Vilko’s heart beats with courage, happiness, and boundless love.

Out of the deepest love for Viliam and the desire to give him a chance for a life like other children have, his parents founded a non‑profit organization called the Inštitút Myológie, n.o. Its mission is to connect leading scientists and clinicians from around the world and to support the development of specialized treatment made specifically for Vily. At the same time, they dream of turning the institute into an open platform – a place where experts from different fields of science and technology can join forces, united by one shared goal: helping patients with neuromuscular diseases and bringing them new hope for better life.

How does it impact Viliam's life?

Vilko lives with a rare form of Duchenne muscular dystrophy. It’s a genetic disease due to which his body cannot produce dystrophin—a vital protein that keeps muscles strong and healthy. Without it, the muscles gradually weaken and break down. The first signs usually appear in the muscles of the legs, hips, and shoulders. Over time, children lose the strength to walk, run, or stand up on their own.

As the disease progresses, it begins to affect the upper limb function, breathing, and even the heart. Without treatment, most children between the ages of eight and twelve lose the ability to walk independently, and soon after, more serious complications follow. Tragically, this disease eventually leads to failure of the respiratory muscles or the heart, and the children’s lives are cut heartbreakingly short. That’s why we must act as quickly as possible—to give Viliam and children like him a real chance for life.

Is it possible to save Vily?

The moment Vilko’s parents learned about his diagnosis, his father reached out to an international team of scientists and doctors capable of developing a specialized treatment just for him. Today, a group of world‑class experts is working together for Vily’s life—professor Wein from France, a leading specialist in gene therapy; professor Škorvánek from the Faculty of Medicine at UPJŠ in Košice; and associate professor Šoltýsová from Comenius University in Bratislava.

These remarkable experts and their colleagues stand among the best in their fields, and thanks to their knowledge and commitment, there is a real hope that our little Vilko can live a better life than that prescribed by Duchenne. The treatment being developed especially for him could also help other children around the world who share a similar genetic mutation in the future. Our effort, therefore, has the power not only to save one precious boy but also to open new doors for scientific progress and give hope to countless families.

Time is working against Viliam

The first four years of life are critical for children with this disease—and sadly, Vilko is already two years old. Every single day, he’s losing muscle strength that can never be regained. Walking is becoming harder, he falls more often, and each morning it takes him longer to stand up on his own. To save him, the treatment must be given as soon as possible—because every day matters, and every moment counts.

Please, support the development of a life-saving treatment for Vily

The total amount needed to develop the treatment in cooperation with leading scientists and to save Viliam’s life is 3 963 500 eur. Each contribution helps us move this unique therapy from hope to reality.​

First milestone 880 000 €​

• Production of the first dose of the therapeutic product in a specialized facility in the USA and testing its effectiveness on Viliam’s cells and models, carried out by experts from the gene therapy institute in Nantes together with Slovak scientists.​

Second milestone 1 012 886 €

• Safety and Toxicity study of the treatment in a specialized GLP laboratory, together with the manufacturing and validation of the therapeutic product for administration, as well as the preparation of the documentation required to approve a national clinical trial in Slovakia.​

Third milestone 1 800 000 €​

• Production of the final cGMP‑grade dose in a specialized facility in the USA, necessary for administering the treatment to Vilko, along with preparation of essential documentation and certification for use in a clinical trial.​

Fourth milestone 270 614 €.

• Costs associated with conducting the clinical research part itsel, i.e. administration of the treatment and subsequent medical care for Vily – carried out at Louis Pasteur University Hospital in Košice, in cooperation with the Faculty of Medicine at UPJŠ in Košice and professor Wein with his team.​

Every single donation brings us closer to a treatment that can save Viliam’s life and stop this cruel disease. With the help of generous people, there is a real hope to halt its progression and open a path to a brighter future.​

Thank you from the bottom of our hearts for helping us to save Vily.