Clinical outcomes following prescription of Vosoritide in children with Achondroplasia 0-2 years of age

This study will prospectively observe the growth and developmental outcomes of children who are given a daily subcutaneous injection of vosoritide prescribed under the Australian prescribing guidelines, in comparison to age-matched historical controls who did not receive any therapeutic intervention. Outcomes measured will be achondroplasia-related comorbidities, age meeting developmental milestones, functional/quality of life status, skull and brain morphology, growth and body proportions. Assessments completed for prospective participants receiving vosoritide will be those routinely collected as part of standard of care for patients receiving vosoritide therapy. These assessments include: physical examination, weight, growth measurements, vital signs, achondroplasia developmental checklist, adverse events, MRI or sleep studies as clinically indicated, review of any interventions related to achondroplasia management, review of vosoritide administration and management. The only addition to routine assessment is the WeeFIM functional and quality of life measure which will be administered for participants >3 years. The total time to complete all assessments will be approximately one hour. The overall duration of observation will be five years from time of enrolment.