1 nonprofit
Real hope for two children (My Daughter) Fund
My daughter Isis and another child, Seppe, suffer from a rare and severe heart disease (restrictive cardiomyopathy caused by a TNNI3 mutation). Currently, there is no effective treatment available.
Over the past period, alongside my work at ASML, I have been fully committed to finding a solution. This has led to an important breakthrough:
we are now collaborating with a professor in the US who has developed a promising peptide — a molecule based on a natural protein from the human body — that directly targets the underlying disease mechanism.
For the first time, there is real hope on the horizon.
We are currently working together with UMC Utrecht to test this peptide on heart cells derived from Isis and Seppe — a next step that is being made possible in part thanks to earlier support and contributions, including from many of you within ASML.
These living heart cells will allow us to test and compare different treatment approaches, including gene therapy.
However, gene therapy falls into a more complex regulatory category, requiring substantial time and investment. As a result, it is less likely to deliver a treatment within a timeframe that could benefit our children.
The next step is the critical bottleneck:
• preclinical studies to determine dosage and toxicity (including animal studies)
These studies are essential and expensive, but required to move safely toward treatment in patients.
• If these two studies are successful, a unique opportunity opens up:
we can potentially proceed via an accelerated pathway (Named Patient / compassionate use) to treat Isis and Seppe.
• If all goes well, treatment could be possible within ~1.5 years.
Importantly, the impact may extend far beyond these two children:
if this treatment proves successful, it could open the door to a therapy for diastolic heart failure with preserved ejection fraction (HFpEF) — a condition affecting millions of people worldwide for which there is currently no effective solution.
For us, this is not theoretical.
This is currently the only realistic path toward a treatment.
To support this work, we use Stichting Tikkertje, which was originally established to fund gene therapy research and is now also supporting this new and promising approach.
💡 Your ASML donation will directly contribute to enabling these crucial next steps, including the testing on patient-derived heart cells and the required preclinical studies.
We are in a race against time.
Every contribution — no matter how small — helps us move faster.
More information:
www.stichtingtikkertje.nl
