Expanding horizons of achondroplasia treatment: current options and future developments

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Publikace nespadá pod Ekonomicko-správní fakultu, ale pod Lékařskou fakultu. Oficiální stránka publikace je na webu muni.cz.
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FAFÍLEK Bohumil BOSÁKOVÁ Michaela KREJČÍ Pavel

Rok publikování 2022
Druh Článek v odborném periodiku
Časopis / Zdroj Osteoarthritis and Cartilage
Fakulta / Pracoviště MU

Lékařská fakulta

Citace
www https://www.sciencedirect.com/science/article/pii/S1063458421009808
Doi http://dx.doi.org/10.1016/j.joca.2021.11.017
Klíčová slova achondroplasia treatment
Popis 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.
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