Lessons Learned from HERA: the First Alport Syndrome Therapeutic Clinical Trial

被引:0
作者
Weinstock, B. Andre [1 ]
机构
[1] Alport Syndrome Fdn, Scottsdale, AZ 85261 USA
来源
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY | 2024年 / 19卷 / 08期
关键词
Alport syndrome; clinical trial;
D O I
10.2215/CJN.0000000000000506
中图分类号
R5 [内科学]; R69 [泌尿科学(泌尿生殖系疾病)];
学科分类号
1002 ; 100201 ;
摘要
引用
收藏
页码:946 / 948
页数:3
相关论文
共 8 条
[1]   Collagen COL4A3 knockout: A mouse model for autosomal Alport syndrome [J].
Cosgrove, D ;
Meehan, DT ;
Grunkemeyer, JA ;
Kornak, JM ;
Sayers, R ;
Hunter, WJ ;
Samuelson, GC .
GENES & DEVELOPMENT, 1996, 10 (23) :2981-2992
[2]  
Feldman DL., VOIC PAT EXT LED PAT
[3]   A Randomized Controlled Clinical Trial Testing Effects of Lademirsen on Kidney Function Decline in Adults with Alport Syndrome [J].
Gale, Daniel P. ;
Gross, Oliver ;
Wang, Fang ;
Esteban de la Rosa, Rafael Jose ;
Hall, Matthew ;
Sayer, John A. ;
Appel, Gerald ;
Hariri, Ali ;
Liu, Shiguang ;
Maski, Manish ;
Shen, Yuqian ;
Zhang, Qi ;
Iqbal, Sajida ;
Kowthalam, Madhurima Uppara ;
Lin, Julie ;
Ding, Jie .
CLINICAL JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2024, 19 (08) :995-1004
[4]   Prevalence Estimates of Predicted Pathogenic COL4A3-COL4A5 Variants in a Population Sequencing Database and Their Implications for Alport Syndrome [J].
Gibson, Joel ;
Fieldhouse, Rachel ;
Chan, Melanie M. Y. ;
Sadeghi-Alavijeh, Omid ;
Burnett, Leslie ;
Izzi, Valerio ;
Persikov, Anton V. ;
Gale, Daniel P. ;
Storey, Helen ;
Savige, Judy .
JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY, 2021, 32 (09) :2273-2290
[5]   Diagnostic Utility of Exome Sequencing for Kidney Disease [J].
Groopman, E. E. ;
Marasa, M. ;
Cameron-Christie, S. ;
Petrovski, S. ;
Aggarwal, V. S. ;
Milo-Rasouly, H. ;
Li, Y. ;
Zhang, J. ;
Nestor, J. ;
Krithivasan, P. ;
Lam, W. Y. ;
Mitrotti, A. ;
Piva, S. ;
Kil, B. H. ;
Chatterjee, D. ;
Reingold, R. ;
Bradbury, D. ;
DiVecchia, M. ;
Snyder, H. ;
Mu, X. ;
Mehl, K. ;
Balderes, O. ;
Fasel, D. A. ;
Weng, C. ;
Radhakrishnan, J. ;
Canetta, P. ;
Appel, G. B. ;
Bomback, A. S. ;
Ahn, W. ;
Uy, N. S. ;
Alam, S. ;
Cohen, D. J. ;
Crew, R. J. ;
Dube, G. K. ;
Rao, M. K. ;
Kamalakaran, S. ;
Copeland, B. ;
Ren, Z. ;
Bridgers, J. ;
Malone, C. D. ;
Mebane, C. M. ;
Dagaonkar, N. ;
Fellstrom, B. C. ;
Haefliger, C. ;
Mohan, S. ;
Sanna-Cherchi, S. ;
Kiryluk, K. ;
Fleckner, J. ;
March, R. ;
Platt, A. .
NEW ENGLAND JOURNAL OF MEDICINE, 2019, 380 (02) :142-151
[6]   Alport syndrome: a unified classification of genetic disorders of collagen IV α345: a position paper of the Alport Syndrome Classification Working Group [J].
Kashtan, Clifford E. ;
Ding, Jie ;
Garosi, Guido ;
Heidet, Laurence ;
Massella, Laura ;
Nakanishi, Koichi ;
Nozu, Kandai ;
Renieri, Alessandra ;
Rheault, Michelle ;
Wang, Fang ;
Gross, Oliver .
KIDNEY INTERNATIONAL, 2018, 93 (05) :1045-1051
[7]   Polygenic risk alters the penetrance of monogenic kidney disease [J].
Khan, Atlas ;
Shang, Ning ;
Nestor, Jordan G. ;
Weng, Chunhua ;
Hripcsak, George ;
Harris, Peter C. ;
Gharavi, Ali G. ;
Kiryluk, Krzysztof .
NATURE COMMUNICATIONS, 2023, 14 (01)
[8]   Isotopic Nitrogen-15 Labeling of Mice Identified Long-lived Proteins of the Renal Basement Membranes [J].
Liu, Pan ;
Xie, Xinfang ;
Jin, Jing .
SCIENTIFIC REPORTS, 2020, 10 (01)