SOURCE:

LINK FOR CONTRIBUTIONS:

https://givebutter.com/ctnnb1genetherapy‘

In 20 days the clinical trial is proving something once unimaginable:

Just a few weeks ago, a child received — for the first time in history — a gene therapy specifically for CTNNB1 Syndrome, the same condition Jimena has.

This is not an announcement.
This is not a promise.
This is a real clinical trial underway with the highest scientific rigor.

And in just 20 days, his mother, Špela, is already describing something that until now did not exist:

A clear reduction in spasticity. A more relaxed body. More “normal.”

A child who previously had enormous difficulty walking is now practically walking on his own.

In just 20 days!

With all the scientific caution necessary, yes.
But also with a hope that simply wasn’t there before.

It is worth putting this into context — even as I get goosebumps writing this.

Developing a gene therapy through a pharmaceutical company usually costs between 150 and 250 million euros.

Our foundation — non-profit, driven by families, scientists and doctors — has taken this therapy from zero to a real clinical trial with about 5 million.

Not because we’re magical.

But because there is no marketing, no shareholders, no unnecessary bureaucracy.
There is focus, rigor, and an urgency that does not allow distraction.

Now comes the important part.

In 2026 we want to:

– expand the clinical trial to Spain and the United States
– treat more children
– manufacture new lots of the therapy
– advance regulatory processes

And for that, we need more resources.

Not to “research someday.”
But to treat children now.
To avoid slowing down something that has already proven to be possible.

And what is being built here is not only for CTNNB1.
It is a real doorway opening for many other rare diseases that today have no alternatives.

This is not emotional philanthropy.
It is investment in measurable human impact.
It is extreme efficiency applied to one of the greatest medical challenges of our time.

If you are a business leader, investor, or someone capable of supporting projects that deserve to exist, this is one of those moments when stepping in truly changes things.

The therapy is already underway.

The difference between advancing or stopping — now — is purely financial.

P.S. I’ve put a link in the comments for financial support (and there you can see the video of Špela’s son walking alone — it’s moving).

P.S.2 If you share this post, maybe an entrepreneur with the financial ability to revolutionize this process will see it (and tell them it is tax-deductible).