Miracle daily pill to help condition suffered by 10,000 people could soon be available on ...
A MIRACLE pill that’s been shown to help with many of the symptoms of cystic fibrosis (CF) has been approved for thousands of ...
生物通3 天
PNAS：新一代囊性纤维化治疗
细胞膜上的蛋白聚集机制为囊性纤维化提供了新的治疗靶点。 一项来自加拿大病童医院（SickKids）的新研究揭示了细胞膜上蛋白质组织的底层过程，这一发现可能为创新的囊性纤维化治疗方法铺平道路。 囊性纤维化（CF）是一种影响肺部、胰腺和其他器官的遗传性疾病，由囊性纤维化跨膜传导调节蛋白（CFTR）基因的变异引起。目前已知约有700种变异会导致CF，但现有的治疗方法仅针对其中少数几种，且没有一种能够治愈 ...
NAI5006 天
未来十年，这家囊性纤维化药物的领导者将何去何从？
过去的十年对 Vertex Pharmaceuticals（VRTX） 来说是一个转折点。这家生物技术公司于2012年获得了首个囊性纤维化（CF）药物——Kalydeco的批准，并随后获得了另外四种药物的监管批准，成为CF市场的全球领导者。公司还展示了在这一专业领域之外的实力，一年多前获得了一种用于血液疾病的基因编辑疗法的批准，最近又获得了一种疼痛治疗的批准。 Vertex的CF药物组合帮助公司2 ...
百亿大药新星崛起！前赛诺菲、福泰高管联手，IPO新锐挑战“不可 ...
2025年2月10日，美国生物制药公司Sionna Therapeutics宣布完成2.192亿美元首次公开募股（纳斯达克股票代码“SION”）。这是继来自中国的亚盛医药、Metsera和Maze Therapeutics之后又一家抓住IPO窗口期成功登陆纳斯达克的制药公司。
Cystic fibrosis patients could get new 'once-a-day' pill this year
Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...
Science Daily2 天
Newly uncovered mechanism could drive next-gen cystic fibrosis treatments
The Hospital for Sick Children. "Newly uncovered mechanism could drive next-gen cystic fibrosis treatments." ScienceDaily. ScienceDaily, 13 March 2025. <www.sciencedaily.com / releases / 2025 / 03 / ...
Where Will Vertex Pharmaceuticals Be in 10 Years?
Vertex Pharmaceuticals generated more than $11 billion in product revenue last year thanks to its dominance in cystic ...
Zacks Investment Research on MSN20 小时
Vertex Up 25% YTD: Should You Buy, Sell or Hold the Stock?
Vertex Pharmaceuticals Incorporated VRTX stock has plunged 25.3% this year so far against the industry’s 5.4% increase. VRTX ...
McGill University Health Centre3 天
Major funding for research project on cystic fibrosis
A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...
生物通5 天
胎儿 CFTRK464N变异或增中国家庭早产风险：一项重要遗传研究成果
为了深入了解这些潜在的遗传风险，来自浙江大学医学院附属妇产科医院等多个机构的研究人员，开展了一项极具意义的研究。他们将目光聚焦于囊性纤维化跨膜传导调节因子（CFTR）基因，这个基因在人体的呼吸、消化和生殖系统中都发挥着至关重要的作用。一旦它发生突变，就可能引发囊性纤维化（CF）等多种严重疾病。而在 ART 日益普及的背景下，CFTR ...
Technology Networks4 天
Newly Uncovered Protein Clustering Mechanism Could Drive New Cystic Fibrosis Treatments
Studying how proteins cluster and organize themselves at the cell membrane could pave the way for innovative cystic fibrosis ...
BioWorld5 天
Adenine base editing corrects G542X mutation in CFTR in patient organoid models
Cystic fibrosis (CF) is a life-threatening autosomal recessive disease affecting over 160,000 people worldwide. CF is caused by loss-of-function mutations in the CF transmembrane conductance regulator ...