Evaluating the impact of vosoritide on complications of achondroplasia
Abstract
PURPOSE: Impaired endochondral bone growth in people with achondroplasia causes complications that impair function, cause pain, require surgery, and contribute to mortality. Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH) rates. METHODS: Prevalence (medical history + new events post-treatment initiation) and incidence (new/worsening events posttreatment initiation) were calculated for complications across 6 clinical trials (2.5-30 μg/kg/day vosoritide) in children with achondroplasia, stratified by age at treatment initiation (
Fields of Study & Subjects
Connective tissue disorders research
Bone and Dental Protein Studies
Dermatological and Skeletal Disorders
Publication details
- Journal / source
- Genetics in Medicine
- Publisher
- Elsevier BV
- Volume
- 28
- Issue
- 7
- Pages
- 102584–102584
- Publication date
- 17 April 2026
- Language
- EN