Expanding horizons of achondroplasia treatment: current options and future developments
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F67985904%3A_____%2F22%3A00559019" target="_blank" >RIV/67985904:_____/22:00559019 - isvavai.cz</a>
Alternative codes found
RIV/00159816:_____/22:00077646 RIV/00216224:14110/22:00129015
Result on the web
<a href="https://asep.lib.cas.cz/arl-cav/cs/csg/?repo=crepo1&key=49583713651" target="_blank" >https://asep.lib.cas.cz/arl-cav/cs/csg/?repo=crepo1&key=49583713651</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1016/j.joca.2021.11.017" target="_blank" >10.1016/j.joca.2021.11.017</a>
Alternative languages
Result language
angličtina
Original language name
Expanding horizons of achondroplasia treatment: current options and future developments
Original language description
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.
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
10601 - Cell biology
Result continuities
Project
Result was created during the realization of more than one project. More information in the Projects tab.
Continuities
I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace
Others
Publication year
2022
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
Osteoarthritis and Cartilage
ISSN
1063-4584
e-ISSN
1522-9653
Volume of the periodical
30
Issue of the periodical within the volume
4
Country of publishing house
NL - THE KINGDOM OF THE NETHERLANDS
Number of pages
10
Pages from-to
535-544
UT code for WoS article
000820228300006
EID of the result in the Scopus database
2-s2.0-85121664571