Cockayne B Spain Association This is Carlota.
She's 4.
She goes to school, she sings, and in the afternoons she makes bracelets with us. She also has an ultra-rare disease — and a treatment is on the way: the challenge is reaching it in time.
We're her family. This is what we've learned over these two years — and why we believe there's still time.
- Goal
- €2,000,000
- Timeframe
- As soon as possible
- For
- Reaching the first clinical trial
One day she started walking and kept falling
When Carlota started to walk, something caught our attention. She fell often and her movements were a little clumsier than usual.
We thought she just needed more time. But our doubts grew with every step.
So began a long road of appointments, tests and months of uncertainty, searching for an answer no one could give us. Finally, a study of her genome put a name to what was happening: Cockayne syndrome type B.
«It took us months to get someone to name it. And when they finally did, we discovered there was a path: a treatment we have to reach in time.»
Today Carlota is 4
She's cheerful, loving and full of hope. She goes to school, loves singing and playing with her friends. In the afternoons she makes bracelets with us — made as a thank-you to donors, and sent all over Spain.
We want her to keep having afternoons like these.
Why we believe we're in time
We're not scientists. We've spent two years learning, asking and knocking on doors. This is what we've found:
- 1
The therapy is already built
A gene therapy for Cockayne syndrome type B exists. It's not an idea on paper: the vector is made and works in animal models.
- 2
Two teams have been at it for years
The University of Minnesota and Portugal's ABC-RI centre, with over €3.5M already invested by other families and institutions.
- 3
What's missing isn't science
It needs manufacturing to pharmaceutical standards, approval by the FDA and EMA, and the trial to start. It needs money and it needs time.
Where every euro goes
Our goal is to raise 2 million euros to make the leap from the preclinical stage to the start of the clinical trial that could treat Carlota and other Spanish children.
€2,000,000
Funding goal
Timeline: as soon as possible
Almost half the goal is manufacturing the medicine. That's the real bottleneck of the project.
-
Manufacture a GMP batch of the therapy
45 %
Produce the vector to pharmaceutical quality, the only kind that can be given to a person.
-
Final preclinical studies
20 %
Complete the trials the agencies require before moving to people.
-
Launch the clinical trial
20 %
Start the trial in hospitals across the European Union.
-
Regulatory process
15 %
Filing and validation with the FDA and the EMA.
Our commitment to transparency
-
Public accounts
We publish the campaign's accounts regularly and accessibly.
-
Progress reports
We share every step of the research honestly, even when the news isn't good.
-
Every euro, traceable
You'll always know which line your contribution went to.
Your help becomes time for Carlota
We're not selling anything. Every contribution goes straight to the research and, as a thank-you, you get a keepsake handmade by Carlota. The treatment can stop her disease: that's why money and time matter.
How to contribute
- 1
Make your contribution by bank transfer to the account below.
- 2
Want your keepsake? Write to info@elcaminodecarlota.org and we’ll send it with a note from Carlota.
Bank transfer
ES17 0182 4924 1102 0163 0508
Account holder: Asociación Cockayne B España (ACBE) · Reference: your name
Frequently asked questions
Is my donation tax-deductible?
Of course. Your help counts just the same from any country.
Can I contribute from outside Spain?
Of course. Your help counts just the same from any country.
Where exactly does the money go?
To the four lines shown above: preclinical studies, manufacturing the GMP batch, the regulatory process and launching the clinical trial. We publish the accounts.
Will I get a certificate?
What if I can't give money?
Sharing her story helps more than you'd think, and it's free.
Let's talk. We're here
Want to collaborate, suggest something or simply learn more? Write to us.
Send us an email and we’ll get back to you within a day:
