{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,28]],"date-time":"2026-08-28T06:02:45Z","timestamp":1787896965773,"version":"build-2784847793"},"reference-count":52,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2023,5,1]],"date-time":"2023-05-01T00:00:00Z","timestamp":1682899200000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Bioorganic &amp; Medicinal Chemistry"],"published-print":{"date-parts":[[2023,5]]},"DOI":"10.1016\/j.bmc.2023.117275","type":"journal-article","created":{"date-parts":[[2023,4,20]],"date-time":"2023-04-20T17:00:55Z","timestamp":1682010055000},"page":"117275","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":14,"special_numbering":"C","title":["Current and emerging therapies for Achondroplasia: The dawn of precision medicine"],"prefix":"10.1016","volume":"87","author":[{"given":"Etienne","family":"Dardenne","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Noboru","family":"Ishiyama","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tai-An","family":"Lin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew C","family":"Lucas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.bmc.2023.117275_b0005","doi-asserted-by":"crossref","first-page":"335","DOI":"10.1016\/0092-8674(94)90302-6","article-title":"Mutations in the transmembrane domain of FGFR3 cause the most common genetic form of dwarfism, achondroplasia","volume":"78","author":"Shiang","year":"1994","journal-title":"Cell"},{"key":"10.1016\/j.bmc.2023.117275_b0010","doi-asserted-by":"crossref","first-page":"252","DOI":"10.1038\/371252a0","article-title":"Mutations in the gene encoding fibroblast growth factor receptor-3 in achondroplasia","volume":"371","author":"Rousseau","year":"1994","journal-title":"Nature"},{"key":"10.1016\/j.bmc.2023.117275_b0015","doi-asserted-by":"crossref","first-page":"2385","DOI":"10.1002\/ajmg.a.32485","article-title":"The population-based prevalence of achondroplasia and thanatophoric dysplasia in selected regions of the US","volume":"146A","author":"Waller","year":"2008","journal-title":"Am J Med Genet. Part A"},{"key":"10.1016\/j.bmc.2023.117275_b0020","doi-asserted-by":"crossref","first-page":"43220","DOI":"10.1038\/srep43220","article-title":"Knock-in human FGFR3 achondroplasia mutation as a mouse model for human skeletal dysplasia","volume":"7","author":"Lee","year":"2017","journal-title":"Sci Rep"},{"key":"10.1016\/j.bmc.2023.117275_b0025","doi-asserted-by":"crossref","first-page":"140","DOI":"10.1016\/j.bbadis.2008.11.010","article-title":"Activating Fgfr3 Y367C mutation causes hearing loss and inner ear defect in a mouse model of chondrodysplasia","volume":"1792","author":"Pannier","year":"2009","journal-title":"Biochimica et Biophysica Acta"},{"key":"10.1016\/j.bmc.2023.117275_b0030","doi-asserted-by":"crossref","first-page":"4977","DOI":"10.1242\/dev.125.24.4977","article-title":"Repression of hedgehog signaling and BMP4 expression in growth plate cartilage by fibroblast growth factor receptor 3","volume":"125","author":"Naski","year":"1998","journal-title":"Development"},{"key":"10.1016\/j.bmc.2023.117275_b0035","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1038\/ng0696-233","article-title":"Graded activation of fibroblast growth factor receptor 3 by mutations causing achondroplasia and thanatophoric dysplasia","volume":"13","author":"Naski","year":"1996","journal-title":"Nat Genet"},{"key":"10.1016\/j.bmc.2023.117275_b0040","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aba4226","article-title":"An RNA aptamer restores defective bone growth in FGFR3-related skeletal dysplasia in mice","volume":"13","author":"Kimura","year":"2021","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.bmc.2023.117275_b0045","doi-asserted-by":"crossref","first-page":"4306","DOI":"10.1038\/onc.2009.280","article-title":"Mutant fibroblast growth factor receptor 3 induces intracellular signaling and cellular transformation in a cell type- and mutation-specific manner","volume":"28","author":"di Martino","year":"2009","journal-title":"Oncogene"},{"key":"10.1016\/j.bmc.2023.117275_b0050","article-title":"Computational and functional analyses of HER2 mutations reveal allosteric activation mechanisms and altered pharmacologic effects","author":"Ishiyama","year":"2022","journal-title":"Can Res"},{"key":"10.1016\/j.bmc.2023.117275_b0055","doi-asserted-by":"crossref","first-page":"4081","DOI":"10.1128\/MCB.16.8.4081","article-title":"Profound ligand-independent kinase activation of fibroblast growth factor receptor 3 by the activation loop mutation responsible for a lethal skeletal dysplasia, thanatophoric dysplasia type II","volume":"16","author":"Webster","year":"1996","journal-title":"Mol Cell. Biol"},{"key":"10.1016\/j.bmc.2023.117275_b0060","doi-asserted-by":"crossref","first-page":"935","DOI":"10.1086\/508433","article-title":"A novel mutation in FGFR3 causes camptodactyly, tall stature, and hearing loss (CATSHL) syndrome","volume":"79","author":"Toydemir","year":"2006","journal-title":"Am J Human Genet"},{"key":"10.1016\/j.bmc.2023.117275_b0065","doi-asserted-by":"crossref","first-page":"959","DOI":"10.1002\/humu.22597","article-title":"A novel homozygous mutation in FGFR3 causes tall stature, severe lateral tibial deviation, scoliosis, hearing impairment, camptodactyly, and arachnodactyly","volume":"35","author":"Makrythanasis","year":"2014","journal-title":"Human Mutat"},{"key":"10.1016\/j.bmc.2023.117275_b0070","doi-asserted-by":"crossref","first-page":"1361","DOI":"10.1101\/gad.13.11.1361","article-title":"FGF signaling inhibits chondrocyte proliferation and regulates bone development through the STAT-1 pathway","volume":"13","author":"Sahni","year":"1999","journal-title":"Genes Dev"},{"key":"10.1016\/j.bmc.2023.117275_b0075","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1101\/gad.1179104","article-title":"Constitutive activation of MEK1 in chondrocytes causes Stat1-independent achondroplasia-like dwarfism and rescues the Fgfr3-deficient mouse phenotype","volume":"18","author":"Murakami","year":"2004","journal-title":"Genes Dev"},{"key":"10.1016\/j.bmc.2023.117275_b0080","doi-asserted-by":"crossref","first-page":"911","DOI":"10.1016\/S0092-8674(00)81069-7","article-title":"Fibroblast growth factor receptor 3 is a negative regulator of bone growth","volume":"84","author":"Deng","year":"1996","journal-title":"Cell"},{"key":"10.1016\/j.bmc.2023.117275_b0085","doi-asserted-by":"crossref","first-page":"1656","DOI":"10.3349\/ymj.2015.56.6.1656","article-title":"Limb lengthening in patients with achondroplasia","volume":"56","author":"Park","year":"2015","journal-title":"Yonsei Med J"},{"key":"10.1016\/j.bmc.2023.117275_b0090","first-page":"7","article-title":"Developmental abnormalities of the occipital bone in human chondrodystrophies (achondroplasia and thanatophoric dwarfism)","volume":"13","author":"Marin-Padilla","year":"1977","journal-title":"Birth Defects Orig Article Series"},{"key":"10.1016\/j.bmc.2023.117275_b0095","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1093\/ajcp\/68.1.68","article-title":"Upper cervical myelopathy in achondroplasia","volume":"68","author":"Yang","year":"1977","journal-title":"Am J Clin Pathol"},{"key":"10.1016\/j.bmc.2023.117275_b0100","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1016\/S0030-5898(02)00069-X","article-title":"Lumbar spinal stenosis. Treatment strategies and indications for surgery","volume":"34","author":"Sengupta","year":"2003","journal-title":"The Orthopedic Clinics of North America"},{"key":"10.1016\/j.bmc.2023.117275_b0105","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1159\/000446958","article-title":"Height outcome of recombinant human growth hormone treatment in achondroplasia children: a meta-analysis","volume":"86","author":"Miccoli","year":"2016","journal-title":"Hormone Research in P\u00e6diatrics"},{"key":"10.1016\/j.bmc.2023.117275_b0110","doi-asserted-by":"crossref","first-page":"873","DOI":"10.1007\/s00431-017-2923-y","article-title":"Final adult height in long-term growth hormone-treated achondroplasia patients","volume":"176","author":"Harada","year":"2017","journal-title":"Eur J Pediatr"},{"key":"10.1016\/j.bmc.2023.117275_b0115","doi-asserted-by":"crossref","first-page":"421","DOI":"10.1515\/jpem-2017-0471","article-title":"Growth velocity and biological variables during puberty in achondroplasia","volume":"31","author":"Del Pino","year":"2018","journal-title":"J Pediatric Endocrinol Metabol"},{"key":"10.1016\/j.bmc.2023.117275_b0120","doi-asserted-by":"crossref","first-page":"17300","DOI":"10.1073\/pnas.0407894101","article-title":"Critical roles of the guanylyl cyclase B receptor in endochondral ossification and development of female reproductive organs","volume":"101","author":"Tamura","year":"2004","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.bmc.2023.117275_b0125","doi-asserted-by":"crossref","first-page":"4016","DOI":"10.1073\/pnas.071389098","article-title":"Dwarfism and early death in mice lacking C-type natriuretic peptide","volume":"98","author":"Chusho","year":"2001","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1016\/j.bmc.2023.117275_b0130","doi-asserted-by":"crossref","first-page":"1108","DOI":"10.1016\/j.ajhg.2012.10.014","article-title":"Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia","volume":"91","author":"Lorget","year":"2012","journal-title":"Am J Human Genet"},{"key":"10.1016\/j.bmc.2023.117275_b0135","article-title":"Natriuretic peptide-based novel therapeutics: long journeys of drug developments optimized for disease states","volume":"11","author":"Ichiki","year":"2022","journal-title":"Biology (Basel)"},{"key":"10.1016\/j.bmc.2023.117275_b0140","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1007\/s40262-021-01059-1","article-title":"Pharmacokinetics and exposure-response of vosoritide in children with achondroplasia","volume":"61","author":"Chan","year":"2022","journal-title":"Clin Pharmacokinet"},{"key":"10.1016\/j.bmc.2023.117275_b0145","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1056\/NEJMoa1813446","article-title":"C-type natriuretic peptide analogue therapy in children with achondroplasia","volume":"381","author":"Savarirayan","year":"2019","journal-title":"The New Engl J Med"},{"key":"10.1016\/j.bmc.2023.117275_b0150","doi-asserted-by":"crossref","first-page":"684","DOI":"10.1016\/S0140-6736(20)31541-5","article-title":"Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial","volume":"396","author":"Savarirayan","year":"2020","journal-title":"Lancet"},{"key":"10.1016\/j.bmc.2023.117275_b0155","doi-asserted-by":"crossref","first-page":"1001","DOI":"10.1002\/ajmg.a.61120","article-title":"Height growth velocity during infancy and childhood in achondroplasia","volume":"179","author":"Del Pino","year":"2019","journal-title":"Am J Med Genet. Part A"},{"key":"10.1016\/j.bmc.2023.117275_b0160","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1124\/jpet.119.258251","article-title":"TransCon CNP, a sustained-release C-type natriuretic peptide prodrug, a potentially safe and efficacious new therapeutic modality for the treatment of comorbidities associated with fibroblast growth factor receptor 3-related skeletal dysplasias","volume":"370","author":"Breinholt","year":"2019","journal-title":"J Pharmacol Exp Therapeut"},{"key":"10.1016\/j.bmc.2023.117275_b0165","doi-asserted-by":"crossref","first-page":"4763","DOI":"10.1111\/bcp.15369","article-title":"Phase 1 safety, tolerability, pharmacokinetics and pharmacodynamics results of a long-acting C-type natriuretic peptide prodrug, TransCon CNP","volume":"88","author":"Breinholt","year":"2022","journal-title":"Br J Clin Pharmacol"},{"key":"10.1016\/j.bmc.2023.117275_b0170","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1124\/jpet.114.218560","article-title":"Neutral endopeptidase-resistant C-type natriuretic peptide variant represents a new therapeutic approach for treatment of fibroblast growth factor receptor 3-related dwarfism","volume":"353","author":"Wendt","year":"2015","journal-title":"J Pharmacol Exp Therapeut"},{"key":"10.1016\/j.bmc.2023.117275_b0175","doi-asserted-by":"crossref","first-page":"e0244368","DOI":"10.1371\/journal.pone.0244368","article-title":"In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia","volume":"15","author":"Goncalves","year":"2020","journal-title":"PLoS One1"},{"key":"10.1016\/j.bmc.2023.117275_b0180","doi-asserted-by":"crossref","DOI":"10.3390\/cells10071617","article-title":"Multiple therapeutic applications of RBM-007, an Anti-FGF2 aptamer","volume":"10","author":"Nakamura","year":"2021","journal-title":"Cells"},{"key":"10.1016\/j.bmc.2023.117275_b0185","doi-asserted-by":"crossref","first-page":"R60","DOI":"10.1093\/hmg\/ddv254","article-title":"Receptor tyrosine kinase mutations in developmental syndromes and cancer: two sides of the same coin","volume":"24","author":"McDonell","year":"2015","journal-title":"Human Mol Genet"},{"key":"10.1016\/j.bmc.2023.117275_b0190","doi-asserted-by":"crossref","first-page":"1871","DOI":"10.1172\/JCI83926","article-title":"Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model","volume":"126","author":"Komla-Ebri","year":"2016","journal-title":"J Clin Investigat"},{"key":"10.1016\/j.bmc.2023.117275_b0195","doi-asserted-by":"crossref","first-page":"20915","DOI":"10.1038\/s41598-020-77345-y","article-title":"Evaluation of FGFR inhibitor ASP5878 as a drug candidate for achondroplasia","volume":"10","author":"Ozaki","year":"2020","journal-title":"Scientific Reports"},{"key":"10.1016\/j.bmc.2023.117275_b0200","doi-asserted-by":"crossref","first-page":"4455","DOI":"10.1073\/pnas.96.8.4455","article-title":"A mouse model for achondroplasia produced by targeting fibroblast growth factor receptor 3","volume":"96","author":"Wang","year":"1999","journal-title":"Proc Natl Acade Sci USA"},{"key":"10.1016\/j.bmc.2023.117275_b0205","doi-asserted-by":"crossref","first-page":"14288","DOI":"10.1074\/jbc.C500024200","article-title":"A loss-of-function mutation in natriuretic peptide receptor 2 (Npr2) gene is responsible for disproportionate dwarfism in cn\/cn mouse","volume":"280","author":"Tsuji","year":"2005","journal-title":"J Biol Chem"},{"key":"10.1016\/j.bmc.2023.117275_b0210","doi-asserted-by":"crossref","first-page":"1118","DOI":"10.1158\/2159-8290.CD-12-0210","article-title":"FGFR genetic alterations predict for sensitivity to NVP-BGJ398, a selective pan-FGFR inhibitor","volume":"2","author":"Guagnano","year":"2012","journal-title":"Can Discov"},{"key":"10.1016\/j.bmc.2023.117275_b0215","doi-asserted-by":"crossref","first-page":"1056","DOI":"10.1016\/j.bone.2005.03.006","article-title":"Complementary antagonistic actions between C-type natriuretic peptide and the MAPK pathway through FGFR-3 in ATDC5 cells","volume":"36","author":"Ozasa","year":"2005","journal-title":"Bone"},{"key":"10.1016\/j.bmc.2023.117275_b0220","doi-asserted-by":"crossref","first-page":"F351","DOI":"10.1152\/ajprenal.00232.2013","article-title":"Regulation of renal phosphate transport by FGF23 is mediated by FGFR1 and FGFR4","volume":"306","author":"Gattineni","year":"2014","journal-title":"Am J Physiol. Renal Physiol"},{"key":"10.1016\/j.bmc.2023.117275_b0225","doi-asserted-by":"crossref","first-page":"2342","DOI":"10.1681\/ASN.2007121301","article-title":"FGFR3 and FGFR4 do not mediate renal effects of FGF23","volume":"19","author":"Liu","year":"2008","journal-title":"J Am Soc Nephrol: JASN"},{"key":"10.1016\/j.bmc.2023.117275_b0230","doi-asserted-by":"crossref","first-page":"916","DOI":"10.1016\/j.eururo.2020.08.002","article-title":"Hyperphosphatemia secondary to the selective fibroblast growth factor receptor 1\u20133 inhibitor infigratinib (BGJ398) is associated with antitumor efficacy in fibroblast growth factor receptor 3-altered advanced\/metastatic urothelial carcinoma","volume":"78","author":"Lyou","year":"2020","journal-title":"Eur Urol"},{"key":"10.1016\/j.bmc.2023.117275_b0235","doi-asserted-by":"crossref","unstructured":"Tagawa S, R AS, Dosne A-G, Zhong B, Qi K, Shalaby W et al. 932P - Hyperphosphatemia due to erdafitinib (a pan-FGFR Inhibitor) and anti-tumour activity among patients (Pts) with advanced urothelial carcinoma (UC). Annals of Oncology 30 (2019) v375.","DOI":"10.1093\/annonc\/mdz249.031"},{"key":"10.1016\/j.bmc.2023.117275_b0240","series-title":"Low-dose infigratinib treatment does not lead to changes in phosphorus in preclinical animal studies","author":"Maribel Reyes","year":"2020"},{"key":"10.1016\/j.bmc.2023.117275_b0245","doi-asserted-by":"crossref","first-page":"424","DOI":"10.1158\/2159-8290.CD-14-1029","article-title":"First selective small molecule inhibitor of FGFR4 for the treatment of hepatocellular carcinomas with an activated FGFR4 signaling pathway","volume":"5","author":"Hagel","year":"2015","journal-title":"Can Discov"},{"key":"10.1016\/j.bmc.2023.117275_b0250","doi-asserted-by":"crossref","first-page":"P141","DOI":"10.1158\/1535-7163.TARG-21-P141","article-title":"Abstract P141: preclinical characterization of LOX-24350, a highly potent and isoform-selective FGFR3 inhibitor","volume":"20","author":"Ballard","year":"2021","journal-title":"Mol Can Therapeut"},{"key":"10.1016\/j.bmc.2023.117275_b0255","doi-asserted-by":"crossref","first-page":"P246","DOI":"10.1158\/1535-7163.TARG-21-P246","article-title":"Abstract P246: Discovery and characterization of selective, FGFR1 sparing, inhibitors of FGFR2\/3 oncogenic mutations for the treatment of cancers","volume":"20","author":"Dardenne","year":"2021","journal-title":"Mol Canc Therapeut"},{"key":"10.1016\/j.bmc.2023.117275_b0260","doi-asserted-by":"crossref","unstructured":"Goyal L et al. Abstract P02-02: First results of RLY-4008, a potent and highly selective FGFR2 inhibitor in a first-in-human study in patients with FGFR2-altered cholangiocarcinoma and multiple solid tumors. Mol Can Therapeut. 2021. 20(12_Supplement): p. P02-02-P02-02.","DOI":"10.1158\/1535-7163.TARG-21-P02-02"}],"container-title":["Bioorganic &amp; Medicinal Chemistry"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0968089623001232?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0968089623001232?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,19]],"date-time":"2025-10-19T01:22:21Z","timestamp":1760836941000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0968089623001232"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,5]]},"references-count":52,"alternative-id":["S0968089623001232"],"URL":"https:\/\/doi.org\/10.1016\/j.bmc.2023.117275","relation":{},"ISSN":["0968-0896"],"issn-type":[{"value":"0968-0896","type":"print"}],"subject":[],"published":{"date-parts":[[2023,5]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Current and emerging therapies for Achondroplasia: The dawn of precision medicine","name":"articletitle","label":"Article Title"},{"value":"Bioorganic & Medicinal Chemistry","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.bmc.2023.117275","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 Elsevier Ltd. All rights reserved.","name":"copyright","label":"Copyright"}],"article-number":"117275"}}