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Ongoing Research into Achondroplasia

Ongoing research into impaired endochondral bone growth4

Current treatment does not address the underlying cause of achondroplasia and is limited to management of complications. However, many different approaches to addressing excessive FGFR3 activity have been proposed and are under clinical trial investigation.1-3

None of these approaches has been determined to be safe or effective or approved for use.

References:

  1. Högler W, Ward LM. New developments in the management of achondroplasia. Wien Med Wochenschr. 2020;170(5-6):104-111.
  2. Ireland PJ, Pacey V, Zankl A, Edwards P, Johnston LM, Savarirayan R. Optimal management of complications associated with achondroplasia. Appl Clin Genet. 2014;7:117-125. Published online June 24, 2014.
  3. Wright MJ, Irving MD. Clinical management of achondroplasia. Arch Dis Child. 2012;97(2):129-134.
  4. Laederich MB, Horton WA. Achondroplasia: pathogenesis and implications for future treatment. Curr Opin Pediatr. 2010;22(4):516-523.

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