When our sons Tom (8) and Lev (5) were diagnosed with Friedreich’s Ataxia (FA) two years ago, we were suddenly faced with a fight we never expected. FA is a rare, progressive, and for now incurable disease that attacks the nervous system and heart, stealing balance, movement, and even the heartbeat. This page shares our family’s journey, and the urgent mission we’ve taken on to change the future.
Facing a rare disease diagnosis
– Our fight against FA
When our sons Tom (8) and Lev (5) were diagnosed with Friedreich’s Ataxia (FA) two years ago, we were suddenly faced with a fight we never expected. FA is a rare, progressive, and for now incurable disease that attacks the nervous system and heart, stealing balance, movement, and even the heartbeat. This page shares our family’s journey, and the urgent mission we’ve taken on to change the future.
Tom & Lev
Our oldest son, Tom, once spent hours running, climbing, and playing soccer. Today he can still cross a room, but his balance is failing and his heart is already enlarged – a life-threatening complication of FA. Lev, our youngest, has thankfully not developed any serious symptoms yet, and we work every day to make sure he never has to. The diagnosis was devastating. But in that dark moment we found determination. We began traveling, meeting scientists, doctors, and other families who share the same fight. Their knowledge and courage gave us hope. And a path forward.
Tom & Lev
Our oldest son, Tom, once spent hours running, climbing, and playing soccer. Today he can still cross a room, but his balance is failing and his heart is already enlarged – a life-threatening complication of FA. Lev, our youngest, has thankfully not developed any serious symptoms yet, and we work every day to make sure he never has to. The diagnosis was devastating. But in that dark moment we found determination. We began traveling, meeting scientists, doctors, and other families who share the same fight. Their knowledge and courage gave us hope. And a path forward.
Friedreich’s Ataxia (FA)
Friedreich’s Ataxia is rare but serious. In a country like Sweden, only about two new cases are diagnosed each year, yet an estimated 40,000 people worldwide live with the disease –most showing symptoms in childhood.
Friedreich’s Ataxia (FA)
Friedreich’s Ataxia is rare but serious. In a country like Sweden, only about two new cases are diagnosed each year, yet an estimated 40,000 people worldwide live with the disease –most showing symptoms in childhood.
Building a gene therapy program
Gene therapy has already transformed the outlook for several rare diseases. FA is a strong candidate for this breakthrough: by delivering a healthy FXN gene to affected cells, the treatment could slow or even reverse the disease.
When we met Dr. Ryan Butler and Dr. Steven Gray at UT Southwestern Medical Center, our mission became clear: launch a gene therapy program for children with FA. Together with FA experts Dr. Marek and Dr. Jill Napierala, we now have a world-class team working toward a cure.
”This is not just research – This is a lifeline”
Why Time Matters
- 10,000+ rare diseases exist
- 400 million people are affected
- 95% lack treatment
- 3,000+ children die each day
For children with FA, every day counts.
Building a gene therapy program
Gene therapy has already transformed the outlook for several rare diseases. FA is a strong candidate for this breakthrough: by delivering a healthy FXN gene to affected cells, the treatment could slow or even reverse the disease.
When we met Dr. Ryan Butler and Dr. Steven Gray at UT Southwestern Medical Center, our mission became clear: launch a gene therapy program for children with FA. Together with FA experts Dr. Marek and Dr. Jill Napierala, we now have a world-class team working toward a cure.
”This is not just research – This is a lifeline”
Why Time Matters
- 10,000+ rare diseases exist
- 400 million people are affected
- 95% lack treatment
- 3,000+ children die each day
For children with FA, every day counts.
Support the research
Your support makes the gene-therapy program possible and gives hope to families everywhere.
With deepest gratitude,
Zardasht, Helena, Tom & Lev
Support the research
Your support makes the gene-therapy program possible and gives hope to families everywhere.
With deepest gratitude,
Zardasht, Helena, Tom & Lev