Mirdametinib treatment for the osteofibrous dysplasia RASopathy

The goal of this study was to investigate the molecular response of primary cells cultured from the pseudarthrosis sites of Osteofibrous Dysplasia patients following 24h treatment with vehicle (DMSO), capmatinib (10uM, Catalog# S2788, Selleck Chemicals, USA), selumetinib (5uM, Catalog# S1008, Selleck Chemicals, USA), or mirdametinib (100nM, Catalog# S1036, Selleck Chemicals, USA). Transcriptional changes were Overall design: Primary cells were cultured from n=5 subjects in two different experiments. In experiment 1, cells were treated with vehicle, capmatinib, or selumetinib. In experiment 2, cells were treated with vehicle or mirdmetinib. Data from both experiments are provided. Gene expression was evaluated by RNA-sequencing with data analyzed using Partek Flow.