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Novel therapies for growth disorders

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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.

What is Known:

• Recombinant human growth hormone has been the mainstay of treatment for children with short stature for years.

• Such therapy is not always effective based on the underlying diagnosis (e.g achondroplasia, Turner syndrome). Compliance with daily injections is challenging and can directly affect efficacy.

What is New:

• Recent development of long-acting growth hormone regimens and oral secretagogues can overcome some of these barriers, however several limitations need to be taken into consideration.

• Newer therapies for achondroplasia, and other rare forms of skeletal dysplasias introduce us to a new era of targeted therapies for children with short stature. Clinicians ought to be aware of pitfalls and caveats before introducing these novel therapies to every day practice.

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Author information

Authors and Affiliations

  1. Division of Endocrinology, Children’s National Hospital, Washington, DC, USA

    Despoina M. Galetaki, Nadia Merchant & Andrew Dauber

  2. Department of Pediatrics, George Washington School of Medicine, Washington, DC, USA

    Nadia Merchant & Andrew Dauber

Authors
  1. Despoina M. Galetaki
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  2. Nadia Merchant
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  3. Andrew Dauber
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Contributions

All authors contributed to the conception of this review article. Literature search was conducted by DG, and reviewed by AD. The first draft of the manuscript was written by DG and NM, and was edited and reviewed by AD. All authors commented on previous versions of the manuscript. All authors read and approved the final manuscript.

Corresponding author

Correspondence to Andrew Dauber.

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The authors declare no competing interests.

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Communicated by Peter de Winter

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Galetaki, D.M., Merchant, N. & Dauber, A. Novel therapies for growth disorders. Eur J Pediatr 183, 1121–1128 (2024). https://doi.org/10.1007/s00431-023-05239-y

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