The revolution in the treatment of genetic skeletal disorders: precision therapy in practice
Over the past 15 years, the treatment of genetic skeletal disorders has evolved from purely symptomatic, to the emergence of several precision therapies that promise to change the health outcomes of children affected by these conditions. We have led this knowledge transformation, enabled and fueled by the genomics revolution. This Grand Round will update the progress on the precision treatment of genetic skeletal disorders that sets a template for the better treatment of many other rare genetic conditions.