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@article{1818487,
author = {Fafílek, Bohumil and Bosáková, Michaela and Krejčí, Pavel},
article_location = {Oxford},
article_number = {4},
doi = {http://dx.doi.org/10.1016/j.joca.2021.11.017},
keywords = {achondroplasia treatment},
language = {eng},
issn = {1063-4584},
journal = {Osteoarthritis and Cartilage},
title = {Expanding horizons of achondroplasia treatment: current options and future developments},
url = {https://www.sciencedirect.com/science/article/pii/S1063458421009808},
volume = {30},
year = {2022}
}
TY - JOUR
ID - 1818487
AU - Fafílek, Bohumil - Bosáková, Michaela - Krejčí, Pavel
PY - 2022
TI - Expanding horizons of achondroplasia treatment: current options and future developments
JF - Osteoarthritis and Cartilage
VL - 30
IS - 4
SP - 535-544
EP - 535-544
PB - Elsevier
SN - 10634584
KW - achondroplasia treatment
UR - https://www.sciencedirect.com/science/article/pii/S1063458421009808
N2 - Activating mutations in the FGFR3 receptor tyrosine kinase lead to most prevalent form of genetic dwarfism in humans, the achondroplasia. Many features of the complex function of FGFR3 in growing skeleton were characterized, which facilitated identification of therapy targets, and drove progress toward treatment. In August 2021, the vosoritide was approved for treatment of achondroplasia, which is based on a stable variant of the C-natriuretic peptide. Other drugs may soon follow, as several conceptually different inhibitors of FGFR3 signaling progress through clinical trials. Here, we review the current achondroplasia therapeutics, describe their mechanisms, and illuminate motivations leading to their development. We also discuss perspectives of curing achondroplasia, and options for repurposing achondroplasia drugs for dwarfing conditions unrelated to FGFR3. (C) 2021 Osteoarthritis Research Society International.
ER -
FAFÍLEK, Bohumil, Michaela BOSÁKOVÁ and Pavel KREJČÍ. Expanding horizons of achondroplasia treatment: current options and future developments. \textit{Osteoarthritis and Cartilage}. Oxford: Elsevier, 2022, vol.~30, No~4, p.~535-544. ISSN~1063-4584. Available from: https://dx.doi.org/10.1016/j.joca.2021.11.017.
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