Novel therapies for growth disorders

被引：10

作者：

Galetaki, Despoina M. ^{[1
]}

Merchant, Nadia ^{[1
,2
]}

Dauber, Andrew ^{[1
,2
]}

机构：

[1] Childrens Natl Hosp, Div Endocrinol, Washington, DC 20010 USA

[2] George Washington Sch Med, Dept Pediat, Washington, DC 20052 USA

来源：

EUROPEAN JOURNAL OF PEDIATRICS | 2024年 / 183卷 / 03期

关键词：

Growth hormone (GH); Long-acting GH; Short stature; Growth hormone deficiency; Somatrogon; Somapacitan; Lonapegsomatropin; Achondroplasia; IGF-1; CNP; TransCon CNP; Vosoritide; Infigratinib; LUM201; Burosumab Carbamazepine; GH-DEFICIENT PATIENTS; HORMONE THERAPY; FACTOR-I; CHILDREN; ADHERENCE; OUTCOMES; MOD-4023; PHASE-2; SAFETY;

D O I：

10.1007/s00431-023-05210-x

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

As we continue to understand more about the complex mechanism of growth, a plethora of novel therapies have recently been developed that aim to address barriers and optimize efficacy. This review aims to explore these novel therapies and provide a succinct review based on the latest clinical studies in order to introduce clinicians to therapies that will soon constitute the future in the field of short stature. Conclusion: The review focuses on long-acting growth hormone formulations, a novel growth hormone oral secretagogue, novel treatments for children with achondroplasia, and targeted therapies for rare forms of skeletal dysplasias.

引用

页码：1121 / 1128

页数：8

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