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Help Build a Treatment for Lucas
There is no existing treatment, we are funding the work to create one.
Why Your Donation Matters
Lucas is living with an ultra-rare genetic condition caused by a mutation in the RBM28 gene — with only two known cases in the world.
There is no treatment, no clinical pathway, and no research currently in place.
Your donation is not just support — it is what makes this work possible.
What Your Donation is Funding
Your support directly funds a coordinated research and therapy development strategy, including:
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Gene therapy development (AAV-based approach)
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Disease biology research to understand how RBM28 impacts the body
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Patient-derived cell studies, including international research collaborations
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Drug screening and repurposing efforts
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Pre-clinical studies and validation
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Regulatory preparation and pathway development
This work is being carried out across multiple institutions to move toward a real treatment as efficiently and responsibly as possible.
What your Donation is NOT For
Lucas is living with an ultra-rare genetic condition caused by a mutation in the RBM28 gene — with only two known cases in the world.
There is no treatment, no clinical pathway, and no research currently in place.
Your donation is not just support — it is what makes this work possible.
Why This Matters
Where We Are Now
This is bigger than Lucas.
Because this condition is so rare, no one else is funding this work.
Without action, there is no path forward.
By supporting this effort, you are helping create a treatment where none exists — not just for Lucas, but for future children who may face the same diagnosis.