軟骨發育不全是骨骼發育不良的最常見類型，全世界范圍內約有25萬人受其影響。軟骨發育不全可導致嚴重的骨骼并發癥和合并癥。例如，骨骼發育異常可以導致睡眠呼吸暫停，下腰段脊髓受沖擊導致慢性背部和腿部疼痛，以及頸髓壓迫導致嬰兒猝死等。咽鼓管異常引起的慢性耳部感染可導致聽力喪失和語言發育遲緩^{[6]<\/sup>。<\/p> \n}

軟骨發育不全是由成纖維細胞生長因子受體3（FGFR3）基因中的常染色體顯性激活突變引起的，該突變導致FGFR3和CNP信號通路的作用失衡。臨床前和臨床數據表明，CNP通路刺激生長發育。補充CNP可抵消FGFR3下游突變的影響，從而可以促進骨骼的生長^{[7]<\/sup>。<\/p> \n}

Ascendis Pharma正在應用其創新的TransCon技術平臺，建立一家全球領先的一體化生物制藥公司，專注于為患者的生活帶來有意義的轉變。在以患者、科學和熱忱為核心價值的引領下，公司依托其TransCon技術平臺潛心開創全新、一流的治療方法。Ascendis總部位于丹麥哥本哈根。了解更多關于Ascendis Pharma 和TransCon技術平臺的相關信息，請訪問Ascendis官網https:\/\/www.ascendispharma.com\/<\/a> <\/p> \n

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久久久亚洲欧洲日产国码二区
[1]. Ascendis Pharma: Once-Weekly TransCon? CNP Achieved Primary Efficacy Objective, with Superiority at 100 µg\/kg\/week, in Ascendis Pharma's Phase 2 ACcomplisH Trial of Children with Achondroplasia Aged 2 to 10 Years https:\/\/investors.ascendispharma.com\/news-releases\/news-release-details\/once-weekly-transcontm-cnp-achieved-primary-efficacy-objective<\/a> <\/span><\/p> <\/td> \n <\/tr> \n
[2]. 關于公布第二批罕見病目錄的通知https:\/\/www.gov.cn\/zhengce\/zhengceku\/202309\/content_6905273.htm<\/a><\/span><\/p> <\/td> \n <\/tr> \n
[3]. Ascendis Pharma Provides Update on Vision 3x3 Strategic Roadmap at 41st Annual J.P. Morgan Healthcare Conference https:\/\/investors.ascendispharma.com\/news-releases\/news-release-details\/ascendis-pharma-provides-update-vision-3x3-strategic-roadmap-0<\/a> <\/span><\/p> <\/td> \n <\/tr> \n
[4]. Ascendis Pharma Reports Second Quarter 2023 Financial Results<\/span><\/p> <\/td> \n <\/tr> \n
https:\/\/investors.ascendispharma.com\/news-releases\/news-release-details\/ascendis-pharma-reports-second-quarter-2023-financial-results<\/a><\/span><\/p> <\/td> \n <\/tr> \n
[5]. 國新辦舉行"權威部門話開局"系列主題新聞發布會介紹"強化藥品監管切實保障人民群眾用藥安全"有關情況圖文實錄http:\/\/www.scio.gov.cn\/xwfb\/gwyxwbgsxwfbh\/wqfbh_2284\/49421\/50109\/wz50111\/202307\/t20230724_729059.html<\/a><\/span><\/p> <\/td> \n <\/tr> \n
[6]. Ireland PJ, Pacey V, et al. Appl Clin Genet. 2014;7:117–25.<\/span><\/p> <\/td> \n <\/tr> \n
[7]. Horton WA, et al. Lancet. 2007;370(9582):162–172.<\/span><\/p> <\/td> \n <\/tr> \n <\/tbody> \n <\/table> \n<\/div> \n
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