New treatment strategies for Fibrodysplasia Ossificans Progressiva

This research project proposes to fully develop a method that includes High Throughput Screening of small molecules, characterization and validation assays of selected compounds and synthesis of new compounds with the aim to achieve a list of compounds suitable for tretament of Fibrodysplasia Ossificans Progressiva (FOP). In the case of compounds already approved for clinical use of other diseases, we expect to obtain reliable results that provide an indication for their use in FOP. In the case of new compounds, we expect to provide proof of concept that they can undergo further steps necessary for possible clinical use.