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This site is intended for U.S. healthcare professionals only

For Patients & Caregivers

ONGOING RESEARCH INTO ACHONDROPLASIA

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.

Ongoing research into impaired endochondral bone growth4

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impaired endochondral bone growth diagram
1
Chemical inhibitors to reduce FGFR3 tyrosine kinase activity
2
Antibodies to block receptor activation
3
Exogenous CNP to enhance CNP-mediated antagonism of downstream signals
4
RNAi to reduce FGFR3 production
5
Hsp90 inhibitors to induce degradation of activated receptor
6
Agents to disrupt direct nuclear signaling of FGFR3

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.