Hi, our names are Sophia & Victor, and we are the proud parents of our son, Lucas—a joyful, resilient little boy who is facing challenges no child should have to face.
Every parent dreams of watching their child grow into independence — taking their first steps, running freely, climbing stairs, speaking with confidence, choosing their own path.
For Lucas, much of that future is still unknown.
Lucas lives with an ultra-rare genetic condition to the gene RBM28 that affects his entire body. It is so rare, there is no information about how it progresses, what his future may look like, or how much independence he may one day have. Some questions are especially hard to carry — we do not know whether his life expectancy is limited, or whether he will reach adulthood. That uncertainty stays with us every day.
Right now, Lucas’s limitations affect nearly every moment of his life.
He crawls or is carried to the bathroom. He must be lifted onto the toilet. In the mornings, he pushes his body with everything he has just to stand upright long enough for his pants to be pulled down. His core is too weak to hold himself steady on a regular seat.
Stairs are particularly challenging. He can crawl up them, but it is dangerous — he slips. He cannot crawl down at all and must always be carried. Walking takes enormous effort. With a hand held, he can manage short distances, but it is slow and exhausting. A walker helps, but spasticity in his lower limbs causes his ankles to kick inward, often making him trip. When he becomes excited, the spasticity intensifies, pushing him onto his toes and causing him to fall forward without warning.
Lucas’s body does not move the way most bodies do. Twisting to reach or grab something — especially while standing — is extremely difficult. His index finger is weak, so he avoids using it, making it hard to hold smaller or heavier items. Simple movements that other children do without thinking require constant effort, balance, and support.
His speech is limited. While his family understands him, many people do not. He can express simple needs and moments of joy, but communication remains a daily challenge. Dressing himself is not possible without help — even sitting to lift a shirt over his head can cause his body to tip and fall without support.
And yet, Lucas is joyful. He is determined. He wants to do things on his own.
What brings Lucas the most joy — more than anything — is walking.
Every opportunity to stand, to take steps, to move forward on his own fills him with pride. Even when it is hard, even when it takes everything he has, walking makes him feel capable. It gives him a sense of freedom, connection, and possibility that few other things do.
What makes this journey especially heartbreaking is not just what Lucas faces today — it’s what we don’t know about tomorrow.
WHY RESEARCH AND GENE THERAPY MATTER
Gene therapy offers something we have never had before: hope for answers and progress.
In simple terms, gene therapy gives Lucas’s body a chance to grow and function more like yours and mine. It may allow his muscles, balance, coordination, and strength to develop in ways they currently cannot. It offers the possibility of a dramatically improved quality of life — and, most importantly, a chance at greater independence.
Research may also help us understand what lies ahead for Lucas — how his condition may progress, what challenges may come, and what future may be possible for him.
WHY WE ARE ASKING FOR HELP?
The estimated cost to develop gene therapy for Lucas’s condition is approximately $1.2 million USD (about $1.7 million CAD). The first required balance is $213,000 USD, which is needed to begin the next critical steps in this research. The research will not begin until the $213 000 USD is reached.
WHY SO EXPENSIVE?
Getting a clinical trial off the ground costs millions of dollars. For rare diseases, large pharmaceutical companies often walk away—not because the science isn’t promising, but because there isn’t enough profit. That leaves families like ours fighting alone. Without the funding, the clinical trial simply will not happen.
WHAT IS GENE THERAPY?
Gene therapy is a medical approach that aims to treat or correct genetic conditions by addressing the problem at its source—the gene itself.
In many genetic disorders, a gene is not working properly or is missing important instructions. Gene therapy works by delivering a healthy copy of a gene into the body’s cells so they can function the way they are supposed to.
HOW DOES GENE THERAPY WORK?
One of the safest and most widely used delivery methods for gene therapy is something called an AAV (adeno-associated virus) vector.
AAVs are not harmful and do not cause disease. Scientists remove the virus’s original genetic material.The virus is then used as a delivery vehicle to carry a healthy gene into the cell. Once inside the cell, the new gene provides instructions to help the cell work properly.
You can think of AAV as a delivery truck carrying the correct instructions into the body.
IS GENE THERAPY THE ONLY TREATMENT OPTION?
Yes. Unfortunately the current intensive therapy that Lucas does 5 days a week is only to preserve the skill he currently has. Gene therapy is the only treatment that will stop the gene from causing further damage to Lucas’s body. We do not know what this gene can do, but we do know it is progressive in nature, as we know Lucas has regressed and lost skill in the last few years.
Once this gene therapy is developed it can be used for future children with this gene variant. With your help today you wouldn’t just be helping Lucas, but giving other children and families the hope to not hear “there is no cure”. You can be a part of that change. No matter how rare, or how many children are impacted, every single child should have access to life saving treatment.
WHAT CAN YOU DO TO HELP LUCAS?
1. Give if you are able. Every single donation—no matter the amount—brings Lucas one step closer to the treatment he desperately needs.
2. Share Lucas's story far and wide. A simple share can reach the one person who changes everything. Please visit our social media pages at @letscurelucas
3. Send this GoFundMe link to your family, friends, coworkers, and anyone who might be willing to help. Sometimes all it takes is one email to spark hope.
4. Help us make this impossible to ignore. Use any platform, connection, or opportunity you have to help this story travel. If you have ideas, media contacts, or leads, please reach out to [email redacted]
Please follow along on our social media accounts at @letscurelucas to daily updates on his progress.
Every contribution helps move this research forward.
Every share brings us closer to answers.
Thank you for seeing Lucas.
Thank you for walking alongside us.
Every bit of support helps give Lucas more chances to do the thing he loves most — to walk forward into his future.
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**follow along @letscurelucas for updates**
