1）通过单次给药的PK研究，评估人纤维蛋白原在先天性纤维蛋白原减少或缺乏症患者中的药代动力学（PK）特征； 2）观察人纤维蛋白原治疗先天性纤维蛋白原减少或缺乏症患者的有效性和安全性； 3）通过单次给药的PK研究，评估市售人纤维蛋白原的药代动力学（PK）特征，为获得性低纤维蛋白原血症的对照药用量计算提供依据。

开始日期2024-04-12

申办/合作机构

广东丹霞生物制药有限公司

100 项与人纤维蛋白原(广东丹霞) 相关的临床结果

登录后查看更多信息

100 项与人纤维蛋白原(广东丹霞) 相关的转化医学

登录后查看更多信息

100 项与人纤维蛋白原(广东丹霞) 相关的专利（医药）

登录后查看更多信息

项与人纤维蛋白原(广东丹霞) 相关的文献（医药）

2024-08-14·JCEM Case Reports

Growth Hormone Deficiency in an Adolescent With Pseudohypoparathyroidism Type 1B

Article

作者: Jüppner, Harald ; Reyes, Monica ; Topor, Lisa Swartz ; Sasidharan Pillai, Sabitha

AbstractWe report growth hormone (GH) deficiency due to presumed GH releasing hormone (GHRH) resistance in an adolescent with pseudohypoparathyroidism type 1B (PHP1B) due to paternal uniparental disomy of chromosome 20 (patUPD20). A male patient aged 11 years 10 months with obesity and mild developmental delay was found to have hypocalcemia, hyperphosphatemia, and an elevated parathyroid hormone level. History included muscle cramps and leg pain with activity. Examination showed round facies, short stature, and obesity. He was in puberty and bone age was advanced by > 2 years. Detailed genetic workup, including nucleotide sequence analysis of GNAS exons 1-13 and STX16, methylation-sensitive multiplex ligation-dependent probe amplification and analysis of several microsatellite markers for chromosome 20, established the diagnosis of PHP1B due to patUPD20. Muscle cramps and hypocalcemia resolved with calcium carbonate, ergocalciferol, and calcitriol treatment. He was short with linear growth deceleration at around age 13 years. Peak GH concentration was insufficient following stimulation testing. Growth velocity improved with human GH treatment. Although rare, resistance to GHRH can occur in PHP1B and patients with this disorder should be evaluated for GH insufficiency if they present with short stature and reduced growth velocity. Treatment with recombinant human GH may improve growth velocity in such patients.

100 项与人纤维蛋白原(广东丹霞) 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
先天性低纤维蛋白原血症	临床3期	中国	广东丹霞生物制药有限公司	2024-04-12
获得性纤维蛋白原缺乏	临床申请批准	中国	广东丹霞生物制药有限公司	2023-11-21