This is Carlota.
She's 4.
She goes to school, loves to sing, and in the afternoons she makes bracelets with us. She also has Cockayne syndrome type B, an ultra-rare, neurodegenerative disease.
A gene therapy is already in development: we need to raise €2 million to help carry it all the way to a clinical trial.
We are her family. And we are working to get there in time.
- Goal
- €2,000,000
- Raised
- €274,548
- Path travelled
- 14%
Raising funds since 7 August 20262,091 donors
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.
Here we explain what Cockayne syndrome type B is, its symptoms and the treatment that could stop it.
«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.»
Why we believe we're in time
After years searching for a diagnosis, today we have an answer — and a scientific path that opens up new treatment possibilities.
How we got here
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2022 · Carlota is born
Carlota is born in 2022.
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2023 · The first signs
At 15 months she starts walking, and we notice that her gait is different from other children her age. Even so, she keeps reaching her milestones, and paediatric check-ups find nothing that points to a specific disease.
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2023–2025 · Two years searching for answers
For almost two years we keep asking, consulting and looking for an explanation for that different way of walking.
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December 2025 · The first clue
An MRI scan shows an alteration in the white matter. That result changes the course of the search and leads us to a study of her genome.
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April 2026 · The diagnosis
The genetic study confirms it: Cockayne syndrome type B, caused by alterations in the ERCC6 gene. At last we had a name. And we also understood that we needed to know what possibilities existed.
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2026 · Slovenia changes our perspective
We travel to Slovenia for MOCCARE, an international meeting focused on research and treatment of Cockayne syndrome. There we met first-hand the researchers working on a therapy, and a Slovenian family who had already started down that path. We came home with one clear idea: if science can develop a treatment, we have to help it reach the clinical trial — and, one day, Carlota and the children who need it.
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July 2026 · Cockayne B España is born
The Asociación Cockayne B España is founded to drive research forward and speed up the path towards a therapy.
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August 2026 · El Camino de Carlota begins
An initiative of the association to mobilise people, companies and institutions and raise the funding needed to move from research to the clinical trial.
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. Talks with the agencies have already begun.
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.
We want her to keep having afternoons like these.
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 two thirds of the goal is manufacturing the medicine. That's the real bottleneck of the project.
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Manufacture a GMP batch of the therapy
65 %
Produce the vector to pharmaceutical quality, the only kind that can be given to a person.
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Final preclinical studies
5 %
Complete the trials the agencies require before moving to people.
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Launch the clinical trial
18 %
Start the trial in hospitals across the European Union.
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Regulatory process
12 %
Filing and validation with the FDA and the EMA.
Our commitment to transparency
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Public accounts
We publish the campaign's accounts regularly and accessibly.
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Progress reports
We share every step of the research honestly, even when the news isn't good.
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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. The treatment can stop her disease: that's why money and time matter.
How to contribute
💳 Bank card
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💙 Monthly donation
Monthly contributions are the ones that help the most: they let us plan the path.
Straight to the association. Charged today and repeated automatically every month. Cancel any time, with one click.
Walk with Carlota month by month →Do you pay taxes in Spain? Give monthly through Donio and get a donation certificate to deduct up to 80 % of the first 250 € a year. On Donio, choose “Mensual” and tick “Quiero un certificado de donación”.
Give monthly through Donio →Bizum · donation
14716
In the name of: ASOC COCKAYNE B ESPAÑA
In your banking app: Bizum → Donate to an NGO → enter the code.
It’s a Bizum code for NGOs (that’s why it’s shorter than a phone number). Official and secure.
Bank transfer
ES38 3059 0086 0540 8475 6628
Account holder: ASOC COCKAYNE B ESPAÑA · Reference: your name
Want to help every month? Set up a monthly standing order in your online banking with this same IBAN: you choose the amount and can cancel it anytime. Monthly support helps the most, because it lets us plan the road ahead.
Already donated? Leave us your details
Once you've made your Bizum or bank transfer, fill this in so we can thank you and keep you posted on every step.
🧾 Donate and deductIf you pay tax in SpainUp to 80 % tax deduction
50 €
200 €
Opens donio.es in a new tab (Spanish and English). To receive the certificate, tick “I want a donation certificate” when donating. Prefer to give monthly? Choose “Mensual” on Donio: you also get the certificate.
How the tax deduction works
Through Donio, a non-profit foundation that hands over 100 % of what is raised to the campaign and issues the donation certificate for your Spanish tax return. You pay on donio.es by card, Apple Pay or Google Pay.
Spanish income-tax deduction (residents in Spain): 80 % of the first 250 € and 40 % of the rest. Indicative figures, depending on your tax situation.
Frequently asked questions
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.
Is my donation tax-deductible?
Yes, if you donate through Donio: you receive a donation certificate to deduct in Spain up to 80 % of the first 250 € and 40 % of the rest (companies: 40 %). This also applies to monthly donations on Donio. Direct donations to the association by card, Bizum or transfer are not yet deductible.
What if I can't give money?
Sharing her story helps more than you'd think, and it's free.
Share the campaign
Every time you share, you bring Carlota closer to her treatment.
The path kit, ready for your stories
Backgrounds and images with the campaign look, to post on Instagram, WhatsApp or wherever you like. Download, post and tag.
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:
