crinecerfont是一种新型口服的CRF 1拮抗剂,在成年人(NCT03525886 27)和青少年(NCT04045145 28)中,在2阶段研究中降低了关键激素生物标志物。有意义的减少ACTH,17-Hydroxyprogesterone(17OHP)(诊断性肾上腺雄激素前体)和雄激素(关键的肾上腺雄激素)在14天的开放标签治疗后观察到了CRF 1受体抑制剂具有CRF 1受体抑制作用的证明。此外,女性参与者的睾丸激素和雄性参与者的雄性持续酮比率的升高大大降低。27,28,我们报告了Cahtalyst(NCT0490915)的结果,这是一项CAH成年人的3期跨国试验,评估了Crinecerfont改善雄激素控制的功效,并有可能使GC剂量减少到物理范围。本文(NCT04806451)伴随着儿童和青少年(2-17岁)的Crinecerfont(2-17岁)的同伴论文。29
我们的第四季度2024更新提供了三名感兴趣的特工的摘要,并在2024年或2025年提供了预期的批准:vanzacaftor/tezacaftor/tezacaftor/deutivacaftor用于囊性纤维化,Crinecerfont,用于先天性肾上腺肾上腺素增生的crinecerfont,用于cretostostimogene grenadenerepvec carenadenenorepvec bladecvec cancer。还突出了管道中其他精选的药物和生物制剂,包括基因疗法和生物仿制药。将介绍有关阿尔茨海默氏病的最新消息。Other topics this quarter include updates on recent respiratory syncytial virus (RSV) and coronavirus disease (COVID-19) vaccine recommendations from the Centers for Disease Control and Prevention (CDC) and an overview of two Food and Drug Administration (FDA) programs: the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot Program and the Rare Disease Innovation Hub.
编辑委员会的消息Aashima Dabas博士2来自Ispae-Oh Ahila Ahila Ahila Ahila 3新成员,Ispae Fellows,4岁的Ispae Fellows和Winners-糖皮质激素周期性库什库什综合征的诊断和管理Bharani Anand Ramalingam 12诊断角:Genun kerun Keuamr,Drandan niprand in and drana in nipand in arrand in nipand in nipand in arrand in arrand in nipand in niprand in arrand in arad in arad inar dravans in draf inard in ar d d. Dr Pamali Nanda 13 Act GENDER DR Tejasvi Sheshadri 15 Assignment In DSD Drug Corner: Ispad Webinar-Dr Anju Virmani 17 Insulin Thermostability Drug Corner: Crinecerfont Report: FE (Male) With Dr Akshatha A, Dr Vani HN, 22 Hypertension Prof Raghupathy N Learning Pearls-Apps- Dr Archana Hazra Jaivinder, Amarnath, 27首诗,性别肯定护理Raghupathy教授,Anju Virmani,Sukanya,Konpal,Karthik,S Karthik Ideal/ Ideal/最佳报告Anju Virmani博士,34 Sirisha Kusuma,Preeti Singh颁奖典礼和活动