Key points of the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with Achondroplasia.
10.3760/cma.j.cn511374-20251015-00607
- Author:
Hangyu PING
1
,
2
;
Ran DING
;
Cheng HUANG
;
Yue PENG
;
Zikang ZHONG
;
Weiguo WANG
Author Information
1. Center for Hip Preservation in Osteonecrosis and Developmental Dysplasia of the Hip, Department of Orthopaedics, China-Japan Friendship Hospital, Beijing 100029, China. jointwwg@
2. com.
- Publication Type:Journal Article
- MeSH:
Humans;
Achondroplasia/drug therapy*;
Consensus;
Practice Guidelines as Topic;
Child
- From:
Chinese Journal of Medical Genetics
2026;43(1):5-12
- CountryChina
- Language:Chinese
-
Abstract:
Achondroplasia (ACH) is a common inherited skeletal dysplasia (inherited dwarfism) that compromises quality of life across the lifespan. In 2021, vosoritide became the first approved precision therapy for ACH and is now available in more than 40 countries. Compared with prior symptomatic measures, vosoritide has demonstrated favorable efficacy and a reassuring safety profile. Nevertheless, existing international ACH guidelines largely emphasize complication management and symptomatic care, and there is no unified consensus on pharmacologic therapy. To address this gap, an international expert group developed the International Consensus Guidelines for the Implementation and Monitoring of Vosoritide Therapy in Patients with Achondroplasia providing systematic recommendations that span the continuum of care - from initial patient contact and pre-treatment assessment to medication counseling, injection training, and long-term outcome monitoring. These recommendations complement and refine current management and nursing protocols for individuals with ACH and offer practical guidance for clinicians across diverse regions. This article highlights key elements of the guideline to provide evidence-based support and clinical direction for healthcare professionals in China treating children with ACH using vosoritide.
