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

SUN-441 Crinecerfont Shows Favorable Trends in Improving Clinical Outcomes in Children and Adolescents With Classic Congenital Adrenal Hyperplasia: 1-Year Results From the CAHtalyst™ Pediatric Study

SUN-441 Crinecerfont 在改善经典型先天性肾上腺皮质增生症患儿和青少年的临床结局方面显示出良好的趋势:CAHtalyst™ 儿科研究的 1 年结果

Yang, Xue; Yao, Xiao; Qiu, Yinyuan; Sarafoglou, Kyriakie; Lekarev, Oksana; Loechner, Karen; Yang, Ming; Mick, Gail J; Dattani, Mehul T; Bettendorf, Markus; Clemente, Maria; Salerno, Mariacarolina; Farber, Robert H; Chan, Jean L; Rosales, Gelliza B G; Jeha, George S

SUN-415 Crinecerfont Maintains Serum Androstenedione Levels with Reduced Glucocorticoid Doses in Adults with Classic Congenital Adrenal Hyperplasia: 1-Year Results from the CAHtalyst™ Adult Study

SUN-415 Crinecerfont 可在降低糖皮质激素剂量的情况下维持经典型先天性肾上腺皮质增生症成人患者的血清雄烯二酮水平:CAHtalyst™ 成人研究的 1 年结果

Sharma, Pawana; Scotland, Graham; Cruickshank, Moira; Tassie, Emma; Fraser, Cynthia; Burton, Chris; Croal, Bernard; Ramsay, Craig R; Brazzelli, Miriam; Larson, Kajal B; O’Donnell, John; Tanudra, Angela; Cammarata, Anthony P; Rubino, Christopher M; Banerjee, Ritu; Giri, Abhigya; Komarow, Lauren; Souli, Maria; Doernberg, Sarah B; Patel, Robin; Eckburg, Paul B; Clarke, Dave; Long, Jennifer; Chanda, Sanjay; Krause, Kevin M; Easom, Eric; Talbot, George; Rubino, Christopher M; Molga, Angela; Auchus, Richard J; Hamidi, Oksana; Liu, Chienying; Kiefer, Florian W; Rees, Aled; Gambineri, Alessandra; Simeoli, Chiara; Pivonello, Rosario; Jen, Eric; Lin, Vivian H; Sturgeon, Julia; Chan, Jean L; Auchus, Richard J; Sarafoglou, Kyriakie; Geffner, Mitchell E; Pivonello, Rosario; Simeoli, Chiara; Clemente, Maria; Loewen, Gordon; Roberts, Eiry; Farber, Robert H; Chan, Jean L; Sturgeon, Julia; Smith, Evan; Giri, Nagdeep; Auchus, Richard J; Hamidi, Oksana; Bancos, Irina; Saag, Kenneth; Falhammar, Henrik; Srirangalingam, Umasuthan; Calanchini, Matilde; Jen, Eric; Lin, Vivian H; Sturgeon, Julia; Chan, Jean L; Auchus, Richard J; Hamidi, Oksana; Liu, Chienying; Kiefer, Florian W; Rees, Aled; Gambineri, Alessandra; Simeoli, Chiara; Pivonello, Rosario; Jen, Eric; Lin, Vivian H; Sturgeon, Julia; Chan, Jean L

Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines.

在小鼠和猪动物模型中对视网膜下注射的 Cas9 核糖核蛋白进行评估,突显了基因药物治疗转化中的关键考虑因素

Wei Spencer C, Cantor Aaron J, Walleshauser Jack, Mepani Rina, Melton Kory, Bans Ashil, Khekare Prachi, Gupta Suhani, Wang Jonathan, Soares Craig, Kiwan Radwan, Lee Jieun, McCawley Shannon, Jani Vihasi, Leong Weng In, Shahi Pawan K, Chan Jean, Boivin Pierre, Otoupal Peter, Pattnaik Bikash R, Gamm David M, Saha Krishanu, Gowen Benjamin G, Haak-Frendscho Mary, Janatpour Mary J, Silverman Adam P

Systematic identification and characterization of high efficiency Cas9 guide RNAs for therapeutic targeting of ADAR.

系统地鉴定和表征用于ADAR治疗靶向的高效Cas9引导RNA

Gowen Benjamin G, Melton Kory, Leong Weng In, Khekare Prachi, McCawley Shannon, Chan Jean, Boivin Pierre, Jani Vihasi, Cantor Aaron J, Tambe Akshay, Haak-Frendscho Mary, Janatpour Mary J, Wei Spencer C

SAT-418 Crinecerfont Allows for More Physiologic Glucocorticoid Treatment With Reduction of Androstenedione to a Normal Range in Pediatric Patients With Classic Congenital Adrenal Hyperplasia: Post Hoc Analysis of the CAHtalyst™ Pediatric Study

SAT-418 Crinecerfont 可使经典型先天性肾上腺皮质增生症患儿的雄烯二酮水平降至正常范围,从而实现更符合生理的糖皮质激素治疗:CAHtalyst™ 儿科研究的事后分析

Eyal, Hedva; Lodish, Maya; Fechner, Patricia Y; Trinh, Lien N; Kim, Mimi S; Bettendorf, Markus; Salerno, Mariacarolina; Dattani, Mehul T; Rosales, Gelliza B G; Roberts, Eiry; Jeha, George S; Farber, Robert H; Chan, Jean L

ACTM-838, a novel systemically delivered bacterial immunotherapy that enriches in solid tumors and delivers IL-15/IL-15Rα and STING payloads to engage innate and adaptive immunity in the TME and enable a durable anti-tumor immune response.

ACTM-838 是一种新型全身给药的细菌免疫疗法,可在实体瘤中富集,并递送 IL-15/IL-15Rα 和 STING 有效载荷,以激活 TME 中的先天性和适应性免疫,从而实现持久的抗肿瘤免疫反应。

Cron Kyle R, Fang Ping, Pham Oanh, Janes Julie, Brandenburg John, Lu William, Zhu Jonathan, Peterson Bret, Tribble Sara, Kehoe Haixing, Makarova Anastasia, Iannello Alex, Chan Jean, Skoble Justin, He Hailey, Rae Chris, Thanos Christopher D, Udyavar Akshata R

Phase 3 Trial of Crinecerfont in Adult Congenital Adrenal Hyperplasia

Crinecerfont治疗成人先天性肾上腺皮质增生症的3期临床试验

Auchus, Richard J; Hamidi, Oksana; Pivonello, Rosario; Bancos, Irina; Russo, Gianni; Witchel, Selma F; Isidori, Andrea M; Rodien, Patrice; Srirangalingam, Umasuthan; Kiefer, Florian W; Falhammar, Henrik; Merke, Deborah P; Reisch, Nicole; Sarafoglou, Kyriakie; Cutler, Gordon B Jr; Sturgeon, Julia; Roberts, Eiry; Lin, Vivian H; Chan, Jean L; Farber, Robert H

Crinecerfont, a CRF1 Receptor Antagonist, Lowers Adrenal Androgens in Adolescents With Congenital Adrenal Hyperplasia

Crinecerfont,一种 CRF1 受体拮抗剂,可降低先天性肾上腺增生症青少年患者的肾上腺雄激素水平

Newfield, Ron S; Sarafoglou, Kyriakie; Fechner, Patricia Y; Nokoff, Natalie J; Auchus, Richard J; Vogiatzi, Maria G; Jeha, George S; Giri, Nagdeep; Roberts, Eiry; Sturgeon, Julia; Chan, Jean L; Farber, Robert H

702. Efficacy of Fecal Microbiota, Live-jslm After Receipt of Non–Clostridioides difficile Infection Antibiotics: Post Hoc Subgroup Analysis of a Phase 2 Open-Label Trial

702. 接受非艰难梭菌感染抗生素治疗后,活体粪便微生物群的疗效:一项 2 期开放标签试验的事后亚组分析

Kingwara, Leonard; Karanja, Muthoni; Ngugi, Catherine; Kangogo, Geoffrey; Bera, Kipkerich; Kimani, Maureen; Bowen, Nancy; Abuya, Dorcus; Oramisi, Violet; Mukui, Irene; Matsubara, *Yosuke; Mizuno, Keita; Ikeda, Yoshiki; Yakabe, Mitsutaka; Ogawa, Sumito; Bancos, Irina; Falhammar, Henrik; Young, Jacques; Pivonello, Rosario; Russo, Gianni; Stancampiano, Marianna R; Lin, Vivian H; Sturgeon, Julia; Chan, Jean L; Reveles, Kelly R; Gonzales-Luna, Anne J; Golan, Yoav; Alonso, Carolyn D; Guthmueller, Beth; Tan, Xing; Bidell, Monique; Pokhilko, Victoria; Crawford, Carl; Skinner, Andrew M

Crinecerfont Lowers Elevated Hormone Markers in Adults With 21-Hydroxylase Deficiency Congenital Adrenal Hyperplasia

Crinecerfont 可降低患有 21-羟化酶缺乏症先天性肾上腺增生症的成年人体内升高的激素标志物

Auchus, Richard J; Sarafoglou, Kyriakie; Fechner, Patricia Y; Vogiatzi, Maria G; Imel, Erik A; Davis, Shanlee M; Giri, Nagdeep; Sturgeon, Julia; Roberts, Eiry; Chan, Jean L; Farber, Robert H