日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial

Vanzacaftor-tezacaftor-deutivacaftor 用于治疗 6-11 岁囊性纤维化患儿(RIDGELINE 试验 VX21-121-105):一项单臂 3 期试验的分析

Hoppe, Jordana E; Kasi, Ajay S; Pittman, Jessica E; Jensen, Renee; Thia, Lena P; Robinson, Philip; Tirakitsoontorn, Pornchai; Ramsey, Bonnie; Mall, Marcus A; Taylor-Cousar, Jennifer L; McKone, Edward F; Tullis, Elizabeth; Salinas, Danieli B; Zhu, Jiaqiang; Chen, Yih-Chieh; Rodriguez-Romero, Violeta; Sosnay, Patrick R; Davies, Gwyneth

Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials

在12岁及以上囊性纤维化患者中,Vanzacaftor-tezacaftor-deutivacaftor 与 elexacaftor-tezacaftor-ivacaftor 的疗效比较(SKYLINE 试验 VX20-121-102 和 VX20-121-103):两项随机、活性对照、3期试验的结果

Keating, Claire; Yonker, Lael M; Vermeulen, François; Prais, Dario; Linnemann, Rachel W; Trimble, Aaron; Kotsimbos, Tom; Mermis, Joel; Braun, Andrew T; O'Carroll, Mark; Sutharsan, Sivagurunathan; Ramsey, Bonnie; Mall, Marcus A; Taylor-Cousar, Jennifer L; McKone, Edward F; Tullis, Elizabeth; Floreth, Tim; Michelson, Peter; Sosnay, Patrick R; Nair, Nitin; Zahigian, Rachel; Martin, Hannah; Ahluwalia, Neil; Lam, Anna; Horsley, Alexander

Psychometric performance of the CFQ-R-8D compared to the EQ-5D-3L and SF-6D in people with cystic fibrosis

囊性纤维化患者中 CFQ-R-8D 与 EQ-5D-3L 和 SF-6D 的心理测量学性能比较

Mukuria, Clara; Rowen, Donna; Acaster, Sarah; McGarry, Lisa J; Lou, Yiyue; Sosnay, Patrick R; Quittner, Alexandra L

Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials

vanzacaftor-tezacaftor-deutivacaftor 在成人囊性纤维化患者中的安全性和有效性:随机、双盲、对照 II 期试验

Uluer, Ahmet Z; MacGregor, Gordon; Azevedo, Pilar; Indihar, Veronica; Keating, Claire; Mall, Marcus A; McKone, Edward F; Ramsey, Bonnie W; Rowe, Steven M; Rubenstein, Ronald C; Taylor-Cousar, Jennifer L; Tullis, Elizabeth; Yonker, Lael M; Chu, Chenghao; Lam, Anna P; Nair, Nitin; Sosnay, Patrick R; Tian, Simon; Van Goor, Fredrick; Viswanathan, Lakshmi; Waltz, David; Wang, Linda T; Xi, Yingmei; Billings, Joanne; Horsley, Alexander

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

一项双盲、随机、3期临床试验评估了elexacaftor联合tezacaftor和ivacaftor方案治疗F508del纯合突变囊性纤维化患者的疗效和安全性。

Heijerman, Harry G M; McKone, Edward F; Downey, Damian G; Van Braeckel, Eva; Rowe, Steven M; Tullis, Elizabeth; Mall, Marcus A; Welter, John J; Ramsey, Bonnie W; McKee, Charlotte M; Marigowda, Gautham; Moskowitz, Samuel M; Waltz, David; Sosnay, Patrick R; Simard, Christopher; Ahluwalia, Neil; Xuan, Fengjuan; Zhang, Yaohua; Taylor-Cousar, Jennifer L; McCoy, Karen S

Correlating Cystic Fibrosis Transmembrane Conductance Regulator Function with Clinical Features to Inform Precision Treatment of Cystic Fibrosis

将囊性纤维化跨膜电导调节因子功能与临床特征相关联,以指导囊性纤维化的精准治疗

McCague, Allison F; Raraigh, Karen S; Pellicore, Matthew J; Davis-Marcisak, Emily F; Evans, Taylor A; Han, Sangwoo T; Lu, Zhongzhou; Joynt, Anya T; Sharma, Neeraj; Castellani, Carlo; Collaco, Joseph M; Corey, Mary; Lewis, Michelle H; Penland, Chris M; Rommens, Johanna M; Stephenson, Anne L; Sosnay, Patrick R; Cutting, Garry R

Sources of Variation in Sweat Chloride Measurements in Cystic Fibrosis

囊性纤维化患者汗液氯化物测量值变异的来源

Collaco, Joseph M; Blackman, Scott M; Raraigh, Karen S; Corvol, Harriet; Rommens, Johanna M; Pace, Rhonda G; Boelle, Pierre-Yves; McGready, John; Sosnay, Patrick R; Strug, Lisa J; Knowles, Michael R; Cutting, Garry R

Bias in CFTR screening panels

CFTR 筛查小组的偏倚

Sosnay, Patrick R; Castellani, Carlo; Penland, Christopher M; Rommens, Johanna M; Lewis, Michelle; Raraigh, Karen S; Corey, Mary; Cutting, Garry R

Benign and Deleterious Cystic Fibrosis Transmembrane Conductance Regulator Mutations Identified by Sequencing in Positive Cystic Fibrosis Newborn Screen Children from California

加州新生儿囊性纤维化筛查阳性儿童的测序结果显示,存在良性和有害的囊性纤维化跨膜传导调节因子突变。

Salinas, Danieli B; Sosnay, Patrick R; Azen, Colleen; Young, Suzanne; Raraigh, Karen S; Keens, Thomas G; Kharrazi, Martin

Benign outcome among positive cystic fibrosis newborn screen children with non-CF-causing variants

囊性纤维化新生儿筛查阳性但携带非致病性变异的儿童预后良好

Salinas, Danieli Barino; Sosnay, Patrick R; Azen, Colleen; Young, Suzanne; Raraigh, Karen S; Keens, Thomas G; Kharrazi, Martin