International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia.
Ravi SavarirayanPenny IrelandMelita IrvingDominic ThompsonInês AlvesWagner A R BaratelaJames BettsMichael B BoberSilvio BoeroJenna BriddellJeffrey CampbellPhilippe M CampeauPatricia Carl-InnigMoira Shang-Mei CheungMartyn CobourneValérie Cormier-DaireMuriel Deladure-MollaMariana Del PinoHeather ElphickVirginia FanoBrigitte FaurouxJonathan GibbinsMari L GrovesLars HagenäsTherese HannonJulie Elizabeth Hoover-FongMorrys KaisermannAntonio Leiva-GeaJuan LlerenaWilliam G MackenzieKenneth MartinFabio MazzoleniSharon McDonnellMaria Costanza MeazziniJosef MileradKlaus MohnikeGeert R MortierAmaka C OffiahKeiichi OzonoJohn A PhillipsSteven PowellYosha PrasadCathleen RaggioPablo RosselliJudith RossiterAngelo SelicorniMarco SessaMary TherouxMatthew ThomasLaura TrespediDavid TunkelColin WallisMichael WrightNatsuo YasuiSvein Otto FredwallPublished in: Nature reviews. Endocrinology (2021)
Achondroplasia, the most common skeletal dysplasia, is characterized by a variety of medical, functional and psychosocial challenges across the lifespan. The condition is caused by a common, recurring, gain-of-function mutation in FGFR3, the gene that encodes fibroblast growth factor receptor 3. This mutation leads to impaired endochondral ossification of the human skeleton. The clinical and radiographic hallmarks of achondroplasia make accurate diagnosis possible in most patients. However, marked variability exists in the clinical care pathways and protocols practised by clinicians who manage children and adults with this condition. A group of 55 international experts from 16 countries and 5 continents have developed consensus statements and recommendations that aim to capture the key challenges and optimal management of achondroplasia across each major life stage and sub-specialty area, using a modified Delphi process. The primary purpose of this first International Consensus Statement is to facilitate the improvement and standardization of care for children and adults with achondroplasia worldwide in order to optimize their clinical outcomes and quality of life.
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