The diagnosis of a rare disease
A Race Against Time. Two years ago, Tom, 8, and Lev, 5 years old were diagnosed with Friedreich’s Ataxia (FA) — a rare, progressive disease that slowly robs children of their ability to walk, speak, and even their heartbeat.
Our oldest son, Tom, loved to run, climb, and play soccer. Today, he can still make it across a room, but his balance is failing him and his heart is already enlarged — a life-threatening complication of FA. Lev has thankfully not developed any serious symptoms yet, and we work every day to make sure he never has to.
FA affects about two new individuals each year in a country like Sweden, with an estimated 40 000 people living with the disease worldwide – most showing symptoms in childhood.
A future already written
When we received the diagnosis, our world shattered. But in our darkest moment, we found the determination to fight. We dedicated our lives to this mission, traveling the world to meet scientists, doctors, and other families.
We promised never to give up
We discovered that gene therapy has transformed the treatment landscape for many neurodegenerative diseases. FA, while complex, is an excellent
When we met Dr. Ryan Butler and Dr. Steven Gray at UTSW, our mission became clear: to establish a gene therapy program for children with FA. With a proven track record and a shared vision, they became our partners in this fight.
Together with leading FA experts Dr. Marek and Dr. Jill Napierala, and pioneers in gene therapy, we launched a program that could change everything.
We wanted to be able to look our children in the eyes and say; We did everything. Everything
This is not just research - This is a lifeline
We are deeply grateful to the Hesselman and Rad families for your unwavering support. A heartfelt thank you to Eva Norell for your generous gift, which played a vital role in making this project a reality–in loving memory of Jan Norell.
We are happy to share that the research is now underway. Every donation goes directly to Dr. Gray and Dr. Butler–fueling our collective mission to advance a groundbreaking gene therapy program for Friedreich’s Ataxia.
Gene therapy has already changed lives. We know it works. We know we can do this — with the right team. But we are racing against time. Every day counts.
- 10,000+ rare diseases exist.
- 400 million people are affected.
- 95% lack treatment.
- 3,000+ children die each day.
We have the opportunity to change that – together.
Let’s rewrite history for these children.
In Stockholm, where we live, there is a beautiful cemetery called Skogskyrkogården. Inscribed on its entrance is:
“Hodie Mihi, Cras Tibi”
Today me, Tomorrow you
A reminder that we owe it to each other to keep fighting.
With deepest gratitude,
Zardasht, Helena, Tom & Lev
