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SAR442501 is a humanized monoclonal antibody derivative developed for the treatment of achondroplasia, the most common form of dwarfism. It specifically targets and binds to fibroblast growth factor receptor 3 (FGFR3), inhibiting its overactive signaling caused by genetic mutations in achondroplasia. By limiting FGFR3 activity, SAR442501 aims to improve and restore normal skeletal growth in affected children. The drug is administered via subcutaneous injection and is currently under investigation in Phase 2 clinical trials for pediatric patients with achondroplasia[1][2][3][6][7].
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