软骨发育不全是骨骼发育不良的最常见类型，全世界范围内约有25万人受其影响。软骨发育不全可导致严重的骨骼并发症和合并症。例如，骨骼发育异常可以导致睡眠呼吸暂停，下腰段脊髓受冲击导致慢性背部和腿部疼痛，以及颈髓压迫导致婴儿猝死等。咽鼓管异常引起的慢性耳部感染可导致听力丧失和语言发育迟缓^{[6]<\/sup>。<\/p> \n}

软骨发育不全是由成纤维细胞生长因子受体3（FGFR3）基因中的常染色体显性激活突变引起的，该突变导致FGFR3和CNP信号通路的作用失衡。临床前和临床数据表明，CNP通路刺激生长发育。补充CNP可抵消FGFR3下游突变的影响，从而可以促进骨骼的生长^{[7]<\/sup>。<\/p> \n}

Ascendis Pharma正在应用其创新的TransCon技术平台，建立一家全球领先的一体化生物制药公司，专注于为患者的生活带来有意义的转变。在以患者、科学和热忱为核心价值的引领下，公司依托其TransCon技术平台潜心开创全新、一流的治疗方法。Ascendis总部位于丹麦哥本哈根。了解更多关于Ascendis Pharma 和TransCon技术平台的相关信息，请访问Ascendis官网https:\/\/www.ascendispharma.com\/<\/a> <\/p> \n

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[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]. 关于公布第二批罕见病目录的通知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]. 国新办举行"权威部门话开局"系列主题新闻发布会介绍"强化药品监管切实保障人民群众用药安全"有关情况图文实录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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