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Novel therapies for growth disorders

Research output: Contribution to journalShort surveypeer-review

Abstract

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. (Table presented.)

Original languageEnglish (US)
Pages (from-to)1121-1128
Number of pages8
JournalEuropean Journal of Pediatrics
Volume183
Issue number3
DOIs
StatePublished - Mar 2024
Externally publishedYes

Keywords

  • Achondroplasia
  • Burosumab Carbamazepine
  • CNP
  • Growth hormone (GH)
  • Growth hormone deficiency
  • IGF-1
  • Infigratinib
  • Lonapegsomatropin
  • Long-acting GH
  • LUM201
  • Short stature
  • Somapacitan
  • Somatrogon
  • TransCon CNP
  • Vosoritide

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health

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