Drug for Argonaute syndrome

We are fundraising to develop treatments for Argonaute syndrome (Ago1 syndrome) and Lessel-Kreienkamp syndrome (Ago2 syndrome)—ultrarare genetic diseases that currently have no cure. AGO Alliance Poland is a non-profit organization born out of love, determination, and the urgent need to help children like my son, Albert.

Albert’s diagnosis changed everything. At three years old, we learned that he had a mutation in the Ago1 gene (Phe180del). This news brought answers but also heartbreak. Argonaute syndrome affects him in so many ways: intellectual and learning disabilities, feeding challenges, epileptic changes, autism, low muscle tone, and a squint. For us, Albert—now six years old—is developmentally like a 1.5-year-old.

Accepting his condition and the prognosis for his future wasn’t easy, but it gave us clarity. Instead of just hoping for a miracle, we chose to act. We created this association to fast-track the development of treatments, not only for Albert but for all children affected by these syndromes.

What We’re Fighting For:

Understanding the Disease: We aim to support scientists in their research to unravel the mechanisms behind Argonaute syndromes.

Drug Repurposing: We’re working to test existing drugs to see if they can improve our children’s development.

Genetic Therapies: We are determined to explore gene therapies that could stop or reverse the progression of the disease.

Supporting Development: We use every available therapy and piece of scientific knowledge to help our kids thrive today.

Time isn’t on our side. These disorders rob our children of the chance to grow up as independent adults. Right now, only around 120 families worldwide share this diagnosis—120 stories of love, uncertainty, struggles, and rare, precious victories.

For us, every step forward is a step toward hope: hope for Albert, hope for other children, and hope for their families. Your support isn’t just about funding research; it’s about giving our kids a chance at a better future—a chance at life.