We don't just tell them,
we help make them happen.
Behind every breakthrough is a child, a family, and a reality. Here, you can meet those Fundación Columbus has walked alongside.
Behind every diagnosis is a life, a family, and a unique way of facing the impossible. Here, you’ll find stories from those who live with this reality every day, and the role we’ve played by their side.
Behind every advance are families still waiting for their chance. Your support can help drive new treatments forward, open new paths, and change the course of more lives.
Irai was born on July 16, 2014. Her parents’ joy was soon overshadowed by a devastating diagnosis: childhood Parkinson’s, an ultra-rare condition for which there was no treatment at the time.
This disease leaves the body and brain disconnected, affecting movement and speech, and preventing Irai from moving, speaking, or even holding up her own little head.
When we learned about her story, we reached out to her family to offer them a possible treatment option we were developing with Professor Krystof Bankiewicz and his team. Two months later, on May 14, 2019, Irai underwent a pioneering gene therapy. And thanks to it, she was reborn.
Today, Irai and her family can lead an almost normal life. Irai walks, talks, and has even learned to ski. But above all,
she has become a symbol of hope for other children living with the same reality.
Michael was born in December 2017, apparently healthy. When he was just six months old, his parents began to notice that something was not right, and after numerous tests he was diagnosed with spastic paraplegia.
This neurodegenerative disease severely and progressively affected his motor functions, turning his life into a countdown.
But giving up was not an option. His parents, Terry and Georgia, decided to turn fear into action and launched their own foundation: CureSPG50. Together with Fundación Columbus, they began driving the development and manufacturing of a gene therapy in the hope of changing the future for children with the same diagnosis.
In March 2022, Michael became the first child in the world to receive the gene therapy Melpida. This treatment marked a turning point, creating a future for Michael and encouraging other families not to give up in their fight against the disease.
Urban was diagnosed with CTNNB1 syndrome when he was just 9 months old. This genetic disorder affects development, movement, and speech. Špela Mirošević, his mother, understood how complex the situation was and decided to take the first step herself.
Drawing on her technical background, she decided to become the driving force behind a search that was not only medical but deeply human: finding an opportunity where there seemed to be none.
In 2021, she founded the CTNNB1 Foundation and began reaching out to scientists around the world to advance a gene therapy that could open up a new horizon. Together with Fundación Columbus, she developed a treatment that now bears the name of the baby who made it possible: Urbagen.
What began as a fight for her son has now become a shared hope for hundreds of families, reminding us that sometimes a mother’s love is stronger than any diagnosis.
Candela
AADC / Infantile Parkinsonism
Candela was just a baby when her family learned that her only hope was a gene therapy. Thanks to the Light for Candela campaign, led by Fundación Columbus, she was able to access that opportunity. What came next was the beginning of a new life, for her and for her entire family.
Alberto
SPG50 / Spastic paraplegia
Alejandro
SPG50 / Spastic paraplegia
For a long time, Alejandro’s future was full of questions. In 2024, he gained access to a gene therapy for SPG50 through a pioneering clinical trial in Europe, made possible by Viralgen, Elpida, Donostia University Hospital, and Fundación Columbus. Sometimes, a single opportunity doesn’t just change one story: it paves the path for those that follow.
Sergio
SPG50 / Spastic paraplegia
For years, Sergio’s family lived with unanswered questions, until they finally learned what was behind his story: SPG50. That answer opened the door to the right gene therapy in 2024. Sometimes, naming what’s happening is also the first step toward changing it.
Thomaz
SPG50 / Spastic paraplegia
Thomaz’s life has been full of signals doctors couldn’t quite read. At 16 months old, he was diagnosed with SPG50, and ever since, between hard work and rehabilitation, he’s been waiting for a gene therapy that could change everything. Because sometimes hope also means moving forward while that opportunity is still on its way.
Ona
SPG50 / Spastic paraplegia
Ona’s premature birth led to an early diagnosis of SPG50. Timely access to treatment opened up a decisive opportunity exactly when she needed it most. Five months after her diagnosis, she received gene therapy: a hopeful step toward her future.
