Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model-Reference-Cited by-同舟云学术

Development of a Weight-Band Dosing Approach for Vosoritide in Children with Achondroplasia Using a Population Pharmacokinetic Model

Published:2024-04-23 Issue:5 Volume:63 Page:707-719
ISSN:0312-5963
Container-title:Clinical Pharmacokinetics
language:en
Short-container-title:Clin Pharmacokinet

Author:

Qi Yulan,Chan Ming Liang,Mould Diane R.,Larimore Kevin,Fisheleva Elena,Cherukuri Anu,Day Jonathan,Savarirayan Ravi,Irving Melita,Bacino Carlos A.,Hoover-Fong Julie,Ozono Keiichi,Mohnike Klaus,Wilcox William R.,Bober Michael B.,Henshaw Joshua

Funder

BioMarin Pharmaceutical

Publisher

Springer Science and Business Media LLC

Link

https://link.springer.com/content/pdf/10.1007/s40262-024-01371-6.pdf

Reference17 articles.

1. Pauli RM. Achondroplasia: a comprehensive clinical review. Orphanet J Rare Dis. 2019;14:1. https://doi.org/10.1186/s13023-018-0972-6.

2. Chan ML, Qi Y, Larimore K, et al. Pharmacokinetics and exposure-response of vosoritide in children with achondroplasia. Clin Pharmacokinet. 2022;61:263–80. https://doi.org/10.1007/s40262-021-01059-1.

3. Vajo Z, Francomano CA, Wilkin DJ. The molecular and genetic basis of fibroblast growth factor receptor 3 disorders: the achondroplasia family of skeletal dysplasias, Muenke craniosynostosis, and Crouzon syndrome with acanthosis nigricans. Endocr Rev. 2000;21:23–39. https://doi.org/10.1210/edrv.21.1.0387.

4. Savarirayan R, Irving M, Harmatz P, et al. Growth parameters in children with achondroplasia: a 7-year, prospective, multinational, observational study. Genet Med. 2022;24:2444–52. https://doi.org/10.1016/j.gim.2022.08.015.

5. Foreman PK, van Kessel F, van Hoorn R, et al. Birth prevalence of achondroplasia: a systematic literature review and meta-analysis. Am J Med Genet A. 2020;182:2297–316. https://doi.org/10.1002/ajmg.a.61787.