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Drugs Fut 2013, 38(4): 263
ISSN 0377-8282
Copyright 2013 Clarivate Analytics
CCC: 0377-8282
DOI: 10.1358/dof.2013.038.04.1928598
Achondroplasia: A view to the future options emerging from the benchside
Narayana, J., Horton, W.A.
Achondroplasia, the most common form of dwarfism in humans, results from mutations that exaggerate signaling of the receptor tyrosine kinase fibroblast growth factor receptor 3 (FGFR-3) in the context of linear bone growth. Recent advances in unraveling its molecular pathogenesis have led to new therapeutic strategies that target FGFR-3 expression, activation, signaling and stability, as well as pathways downstream of this receptor. These strategies involve tyrosine kinase inhibitors, blocking antibodies and peptide and physiological antagonists, RNA interference (RNAi) and chaperone inhibitors. One promising strategy, administration of C-type natriuretic peptide (CNP), has reached clinical trials. Here we examine the physiological basis and nature of these strategies, and also address the challenges of treating growth plate disorders in children.

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