Vosoritide Treatment Guidelines for Achondroplasia: A Consensus Statement

Achondroplasia, the most common form of short-limbed skeletal dysplasia, affects individuals throughout their lifespan, requiring adaptations for daily living. With the approval of vosoritide in 2021, a pharmacological treatment for achondroplasia, a need for clear treatment guidelines emerged. In November 2023, an international expert and patient advocate collaboration developed a Consensus Statement on the use of vosoritide. The guidelines cover the treatment pathway, including initiation, monitoring, and cessation of vosoritide therapy, providing a practical framework for clinicians and health services globally.