💡A huge shout out to all of the authors of this study Tim Dreyer, Jacob Keen, Leah Wells, Mark Hopkinson, Isabel Orriss, Gill Holdsworth and Andy Pitsillides - you are all great! Also many thanks to our funders UCB Pharma for your continued support.

📋 Importance: LGK974 therapy would likely be an option for treating disease early in children. This would potentially avoid risky surgical treatments such as skull decompression surgery.

💊 What We Found: A drug called LGK974 reduced skull and ear bone mass of young mice with sclerosteosis disease, indicating that it effectively limits skeletal overgrowth at sites with severe/fatal pathology.

🔬 What We Did: We screened distinct drugs in cells, and a mouse model of sclerosteosis. This is a disease characterised by skeletal overgrowth, potentially fatal elevated intracranial pressure, and deafness. Sclerosteosis has no pharmaceutical therapy, and is managed only through high-risk surgery.