P209: Real-world healthcare utilization, effectiveness, health-related quality of life, and adherence to vosoritide in children with achondroplasia in the United States

Achondroplasia is a rare skeletal dysplasia that impairs endochondral ossification by inhibiting chondrocyte proliferation and differentiation, often resulting in medical complications and functional impairment. Vosoritide, a C-type natriuretic peptide analog that stimulates endochondral bone growth, is an approved treatment for achondroplasia. Although there is a growing body of real-world evidence, particularly from Europe and Japan, data from the US are limited. Here, we report interim effectiveness and clinical outcome assessments (COA) from children with achondroplasia receiving vosoritide treatment in the observational VIrtual STudy in Achondroplasia for the US (VISTA).