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Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

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Same author

Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia.

The New England journal of medicine·2026
Same author

Evaluating the impact of vosoritide on complications of achondroplasia.

Genetics in medicine : official journal of the American College of Medical Genetics·2026
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Effect of vosoritide on spine morphology in children with achondroplasia: 1-year results from a randomized phase 2 study.

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Effect of vosoritide on genu varum in children with achondroplasia after 1 year in randomized placebo-controlled trials.

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Pathways to Facilitate Early Recognition and Diagnosis of Hypochondroplasia.

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Related Experiment Video

Updated: May 10, 2026

A Rat Tibial Growth Plate Injury Model to Characterize Repair Mechanisms and Evaluate Growth Plate Regeneration Strategies
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Progress in managing children with achondroplasia.

Tashunka Taylor-Miller1, Ravi Savarirayan1,2,3

  • 1Victorian Clinical Genetics Service, Melbourne, Parkville, Victoria, Australia.

Expert Review of Endocrinology & Metabolism
|August 12, 2024
PubMed
Summary

New achondroplasia treatments offer hope for children with this skeletal disorder. Approved therapies like vosoritide and emerging precision medicines aim to improve growth and reduce complications.

Keywords:
Achondroplasiafibroblast growth factor receptor 3 (FGFR3)infigratinibnavepegritidevosoritide

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Area of Science:

  • Skeletal Dysplasias
  • Pediatric Endocrinology
  • Pharmacology

Background:

  • Achondroplasia is a rare genetic skeletal disorder affecting 300,000 worldwide.
  • Historically, treatment for achondroplasia has been limited to symptom management.
  • Recent advancements have introduced effective therapeutic options for affected children.

Purpose of the Study:

  • To review key therapeutic advances in managing pediatric achondroplasia.
  • To discuss approved and investigational precision therapies.
  • To summarize current clinical trial outcomes for these novel treatments.

Main Methods:

  • Review of current literature on achondroplasia therapeutics.
  • Analysis of clinical trial data for vosoritide, navepegritide, and infigratinib.
  • Discussion of the impact of new therapies on patient outcomes.

Main Results:

  • Vosoritide is the first approved drug for achondroplasia, marking a significant therapeutic shift.
  • Emerging therapies include navepegritide and infigratinib, targeting specific pathways.
  • These treatments aim to alter growth trajectories and decrease medical complications.

Conclusions:

  • The introduction of vosoritide represents a paradigm shift in achondroplasia treatment.
  • Precision therapies hold promise for improving growth, functionality, and reducing complications.
  • Early diagnosis and access to targeted therapies from birth are crucial for optimal outcomes.