Shedding New Light: Novel Therapies for Achondroplasia and Growth Disorders

Authors

Nadia Merchant, Division of Endocrinology, Children's National Hospital, Washington, DC 20010, USA; Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, DC 20037, USA. Electronic address: nmerchant@childrensnational.org.
Andrew Dauber, Division of Endocrinology, Children's National Hospital, Washington, DC 20010, USA; Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, DC 20037, USA.

Document Type

Journal Article

Publication Date

10-1-2023

Journal

Pediatric clinics of North America

Volume

70

Issue

5

DOI

10.1016/j.pcl.2023.05.008

Keywords

Achondroplasia; FGFR3; Growth disorders precision medicine; Short stature; Skeletal dysplasia; Treatment

Abstract

Achondroplasia is the most common form of disproportionate severe short stature. Management of achondroplasia requires a multidisciplinary approach and has been largely symptomatic for medical complications and psychosocial implications. Increased understanding of genetic and molecular mechanisms of achondroplasia has led to the development of novel disease-modifying drugs. The current drugs under investigation target the growth plate to stimulate chondrocyte growth and development. These include analogs of C-type natriuretic peptide (CNP), FGFR3-selective tyrosine kinase inhibitors, anti-FGFR3 antibodies, aptamers against FGF2, and soluble forms of FGFR3. Long-term data on the effects of these therapies on medical comorbidities are pending at this time.

APA Citation

Merchant, Nadia and Dauber, Andrew, "Shedding New Light: Novel Therapies for Achondroplasia and Growth Disorders" (2023). GW Authored Works. Paper 3694.
https://hsrc.himmelfarb.gwu.edu/gwhpubs/3694

Department

Pediatrics