Development of a multidisciplinary syllabus to support the education and training of roles in cystic fibrosis care: An ECFS Education initiative [0.03%]
C Smith,H K Chadwick,N Shaw et al.
C Smith et al.
Background: Clarity and consistency of required knowledge and skills is crucial in supporting the development of members of cystic fibrosis (CF) multidisciplinary teams. Syllabi are effective frameworks for delivering a m...
Progression of structural lung disease and lung function in adolescents with cystic fibrosis [0.03%]
囊性纤维化青少年肺结构疾病的发展和肺功能研究
Yuxin Chen,Daan Caudri,Eleni-Rosalina Andrinopoulou et al.
Yuxin Chen et al.
Background: Cystic fibrosis (CF) lung disease begins early in life and progresses throughout childhood into adolescence. Children completing the Australasian CF Bronchoalveolar Lavage (ACFBAL) trial were followed longitud...
Phenotypic and functional characterisation of liver-infiltrating T-cells in a case of CFTR modulator induced drug-induced liver injury [0.03%]
一种由囊性纤维化跨膜转运调节物诱导的药物性肝损伤病例中的肝脏浸润T细胞的表型和功能特征分析
Elsie Clarke,Joshua Gardner,Alanood Howsawi et al.
Elsie Clarke et al.
Background: The introduction of CFTR modulators has been transformative for many people with cystic fibrosis (CF). The drugs are generally well tolerated although adverse reactions such as delayed-type T-cell mediated hyp...
CFTR during pregnancy and adverse drug reactions: A pharmacovigilance disproportionality analysis in VigiBase [0.03%]
囊性纤维化跨膜转导调节因子在妊娠期与药物不良反应的关系：源自VigiBase的信号检测分析
Nazanin Abolhassani,Kim Dao,Roberta Noseda et al.
Nazanin Abolhassani et al.
Background: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have transformed the management of cystic fibrosis (CF), but evidence on their safety during pregnancy remains limited and pregnancy-relate...
Solène Castanier,Ahmad Elbahnsi,Benoit Chevalier et al.
Solène Castanier et al.
The Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulator VX-445 (Elexacaftor) used to treat cystic fibrosis presents both corrector and potentiator activities. This drug binds to a pocket within the CFTR membrane-spanning do...
Continuous glucose monitoring is a better indicator of glucose tolerance than HbA1c in cystic fibrosis [0.03%]
连续血糖监测比糖化血红蛋白更能反映囊性纤维化的葡萄糖耐量状况
Laure Alexandre-Heymann,Quitterie Reynaud,Caroline Grou et al.
Laure Alexandre-Heymann et al.
Background: Systematic screening for cystic fibrosis related diabetes (CFRD) is recommended for all people living with cystic fibrosis (pwCF) from the age of 10. However, adhering to these guidelines is challenging given ...
Correcting CFTR mRNA splicing defects with the plant cytokine kinetin and its analogues [0.03%]
用植物细胞因子玉米素及其类似物纠正CFTR mRNA剪接缺陷
Valeria Rimoldi,Giulia Soldà,Anita Capalbo et al.
Valeria Rimoldi et al.
Background: Cystic Fibrosis (CF) results from CFTR gene mutations, including splicing defects such as the polymorphic TGnTm repeat, which disrupts exon-10 inclusion and contributes to CF monosymptomatic forms. While recen...
Mutations with residual CFTR function are associated with better glucose tolerance and insulin secretion in people with Cystic fibrosis [0.03%]
CFTR残余功能突变与囊性纤维化患者的更好的血糖耐量和胰岛素分泌相关
Fabiana Ciciriello,Andrea Foppiani,Federica Sileo et al.
Fabiana Ciciriello et al.
Background: People with Cystic Fibrosis (pwCF) often exhibit impaired insulin secretion, which may lead to Cystic Fibrosis-Related Diabetes (CFRD). The impact of CF variants in the complex relationship between CFTR channe...
Exposure of dams to Elexacaftor/ Tezacaftor/Ivacaftor during pregnancy and breastfeeding induces reversible alterations in newborn wild type CD-1 mice [0.03%]
孕期和哺乳期接触达美加湿剂Elexacaftor/Tezacaftor/Ivacaftor对野生型CD-1小鼠新生儿的可逆改变
Angelica Squarzoni,Gaia Boschetti,Sine Mandrup Bertozzi et al.
Angelica Squarzoni et al.
Background: We previously demonstrated that Tezacaftor inhibits the enzyme (DEGS) that converts dihydroceramides (dHCer) into ceramides, thus producing accumulation of dHCer in various cells and tissues. We here conducted...
Moving personalized medicine from treatment to prevention - a paradigm shift in genetic conditions [0.03%]
从治疗到预防个性化医学的转变——遗传性疾病观念变革
Raksha Jain
Raksha Jain