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Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study.

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Date

2022-09-16

Authors

Savarirayan, Ravi
Irving, Melita
Harmatz, Paul
Delgado, Borja
Wilcox, William R
Philips, John
Owen, Natalie
Bacino, Carlos A
Tofts, Louise
Charrow, Joel

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Abstract

This study was undertaken to collect baseline growth parameters in children with achondroplasia who might enroll in interventional trials of vosoritide, and to establish a historical control. In this prospective, observational study, participants (≤17 years) underwent a detailed medical history and physical examination and were followed every 3 months until they finished participating in the study by enrolling in an interventional trial or withdrawing. A total of 363 children were enrolled (28 centers, 8 countries). Mean (SD) follow up was 20.4 (15.0) months. In participants This study represents one of the largest datasets of prospectively collected medical and longitudinal growth data in children with achondroplasia. It serves as a robust historical control to measure therapeutic interventions against and to further delineate the natural history of this condition.

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Child
Male
Female
Humans
Child, Preschool
Prospective Studies
Achondroplasia
Body Height

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Keywords

Achondroplasia, Annualized growth velocity, Anthropometrics, Observational, Pediatrics

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