Skeletal dysplasia are a group of heritable conditions caused by abnormalities in the development, growth, and maintenance of the human skeleton. Many of these conditions have had their molecular basis revealed over the past 20 years and, recently, several potential precision therapies have emerged for these conditions.
This talk will overview the new emerging therapies for skeletal dysplasia, highlighting Achondroplasia and Schmid metaphyseal dysplasia as examples of this new paradigm, and discuss the pioneering clinical trials that are evaluating the safety and effectiveness of these new treatments.