六部门联合发布《第二批罕见病目录》，新增86种罕见疾病

关键词

司柏索利单抗 三联疗法 耐药性检测 CRISPR-Cas12a 莫格利珠单抗 胰岛素样生长因子1受体 阿尔茨海默病 替雷利珠单抗 罕见病

今日行业热点

①The Lancet：Efficacy and safety of subcutaneous spesolimab for the prevention of generalised pustular psoriasis flares (Effisayil 2): an international, multicentre, randomised, placebo-controlled trial

临床试验：司柏索利单抗（Spesolimab）用于预防广泛性脓疱性银屑病(Effisayil 2)的有效性和安全性:一项国际、多中心、随机、安慰剂对照试验

DOI: 10.1016/S0140-6736(23)01378-8

②The Lancet Gastroenterology and Hepatology：Trimodality therapy versus perioperative chemotherapy in the management of locally advanced adenocarcinoma of the oesophagus and oesophagogastric junction (Neo-AEGIS): an open-label, randomised, phase 3 trial

三联疗法与围手术期化疗用于局部晚期食管和食管胃结合部腺癌(Neo-AEGIS):一项开放标签、随机、Ⅲ期试验

DOI: 10.1016/S2468-1253(23)00243-1

③The Lancet Microbe：Xpert MTB/RIF Ultra on contaminated liquid cultures for tuberculosis and rifampicin-resistance detection: a diagnostic accuracy evaluation

使用Xpert MTB / RIF Ultra（Ultra）方法对污染液体培养物进行结核和利福平耐药性检测的诊断准确性评价

DOI: 10.1016/S2666-5247(23)00169-6

④The Lancet Microbe：Rapid detection of monkeypox virus using a CRISPR-Cas12a mediated assay: a laboratory validation and evaluation study

利用CRISPR-Cas12a介导的试验快速检测猴痘病毒:一项实验室验证和评估研究

DOI: 10.1016/S2666-5247(23)00148-9

⑤The Lancet Microbe：Mogamulizumab for Treatment of Human T-lymphotropic Virus Type 1-Associated Myelopathy/Tropical Spastic Paraparesis: A Single-Center US-based Series

莫格利珠单抗（Mogamulizumab）用于人类嗜T淋巴细胞病毒1型（HTLV-1）相关脊髓病/热带痉挛性瘫痪:美国单中心系列研究

DOI: 10.1093/cid/ciad281

⑥Journal of the American Heart Association：Insulin‐Like Growth Factor 1 Receptor Deficiency Alleviates Angiotensin II–Induced Cardiac Fibrosis Through the Protein Kinase B/Extracellular Signal‐Regulated Kinase/Nuclear Factor‐κB Pathway

胰岛素样生长因子1受体(IGF-1R)缺乏通过蛋白激酶B（Akt）/细胞外信号调节激酶（ERK）/核因子κB（NF-kB）途径减轻血管紧张素Ⅱ（Angiotensin II）诱导的心脏纤维化

DOI: 10.1161/JAHA.123.029631

⑦乙酰胆碱酯酶抑制剂琥珀八氢氨吖啶片用于轻、中度阿尔茨海默病的III期临床试验已完成揭盲和主要数据的统计分析，达到主要终点

⑧靶向VEGFR2的TKI创新药阿帕替尼用于复发或转移性腺样囊性癌的国际Ⅱ期临床结果积极

⑨替雷利珠单抗单药用于既往接受过含铂化疗的不可切除、局部晚期或转移性食管鳞状细胞癌（ESCC）的适应症已获欧盟委员会（EC）批准

⑩国家卫健委等六部门联合发布《第二批罕见病目录》共纳入86种罕见疾病，目前，我国共有207种罕见病

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