This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor. Data will be collected in two formats for the primary pediatric study population: 1. Participant-mediated access to electronic health records(including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. . 2. Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data. Data will be collected in the following format for the adult cohort: \- Participant-mediated access to electronic health records (including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. The primary study population will include approximately 150 pediatric individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa) however they will only be counted once, based on their treated status at the point of enrollment. The secondary study population will include 20 adolescent and adult participants.
Age range
0 Months – 18 Years
Sex
ALL
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A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Height
Timeframe: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Weight
Timeframe: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Body Mass Index
Timeframe: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Head Circumference
Timeframe: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Annualized growth velocity (AGV)
Timeframe: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Medical diagnoses by age
Timeframe: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Surgical procedures and/or medical interventions by age
Timeframe: Retrospective data and prospective data for up to 5 years starting from time of study enrolment
Changes in physical functioning
Timeframe: every 6 months starting from time of study enrolment up to 5 years
Changes in health-related quality of life (HRQoL)
Timeframe: every 6 months starting from time of study enrolment up to 5 years
Adherence to treatment with VOXZOGO™ (vosoritide), if prescribed
Timeframe: every 6 months starting from time of study enrolment up to 5 years