New Gene Therapy Study Receives Funding

We are thrilled to announce that CASK Research has awarded a significant research grant to fund a new gene therapy programme. This exciting project represents one of the most promising avenues towards a targeted treatment for CASK gene disorders.

About the Research

Gene therapy approaches aim to address the underlying genetic cause of a disorder, rather than just managing symptoms. For CASK disorders, researchers will investigate whether restoring normal CASK protein function can improve neurological outcomes in preclinical models.

Why This Matters

Currently, there is no targeted treatment for CASK disorders. Management focuses on addressing individual symptoms such as epilepsy, feeding difficulties, and developmental delay. A gene therapy approach could potentially address the root cause and transform outcomes for newly diagnosed children.

Timeline

This is early-stage research. Promising results in animal models will need to be replicated and refined before any human trials could be considered. However, this funding represents a vital first step on that journey.

We are grateful to every family, fundraiser, and supporter whose generosity has made this grant possible.