In conversation with Małgorzata Kosla.

Małgorzata Kosla1, Hajrah Khawaja2

  • 1PACS2 Research Foundation, Warsaw, Poland.

The FEBS Journal
|August 22, 2025
PubMed
Summary

Patient advocacy is crucial for ultrarare diseases like PACS2 syndrome. Parent-led foundations drive research and collaboration to find treatments for this severe neurodevelopmental disorder.

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