ASO drug Qalsody (tofersen) targets amyotrophic lateral sclerosis

被引：16
作者：
Jin, Jing [1 ]
Zhong, Xiao-bo [1 ]
机构：
[1] Univ Connecticut, Sch Pharm, Dept Pharmaceut Sci, Storrs, CT 06269 USA
来源：
TRENDS IN PHARMACOLOGICAL SCIENCES | 2023年 / 44卷 / 12期
基金：
美国国家卫生研究院;
关键词：
ANTISENSE OLIGONUCLEOTIDE TOFERSEN; TRIAL;
D O I：
10.1016/j.tips.2023.08.008
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
引用
收藏
页码：1043 / 1044
页数：2
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共 7 条
[1]   Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study [J].
Benatar, Michael ;
Wuu, Joanne ;
Andersen, Peter M. ;
Bucelli, Robert C. ;
Andrews, Jinsy A. ;
Otto, Markus ;
Farahany, Nita A. ;
Harrington, Elizabeth A. ;
Chen, Weiping ;
Mitchell, Adele A. ;
Ferguson, Toby ;
Chew, Sheena ;
Gedney, Liz ;
Oakley, Sue ;
Heo, Jeong ;
Chary, Sowmya ;
Fanning, Laura ;
Graham, Danielle ;
Sun, Peng ;
Liu, Yingying ;
Wong, Janice ;
Fradette, Stephanie .
NEUROTHERAPEUTICS, 2022, 19 (04) :1248-1258
[2]   Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development [J].
Dhuri, Karishma ;
Bechtold, Clara ;
Quijano, Elias ;
Ha Pham ;
Gupta, Anisha ;
Vikram, Ajit ;
Bahal, Raman .
JOURNAL OF CLINICAL MEDICINE, 2020, 9 (06)
[3]   Neurofilament light-chain response during therapy with antisense oligonucleotide tofersen in SOD1-related ALS: Treatment experience in clinical practice [J].
Meyer, Thomas ;
Schumann, Peggy ;
Weydt, Patrick ;
Petri, Susanne ;
Koc, Yasemin ;
Spittel, Susanne ;
Bernsen, Sarah ;
Guenther, Rene ;
Weishaupt, Jochen H. ;
Dreger, Marie ;
Kolzarek, Felix ;
Kettemann, Dagmar ;
Norden, Jenny ;
Boentert, Matthias ;
Vidovic, Maximilian ;
Meisel, Christian ;
Muench, Christoph ;
Maier, Andre ;
Koertvelyessy, Peter .
MUSCLE & NERVE, 2023, 67 (06) :515-521
[4]   Special Section on Pharmacokinetics and ADME of Biological Therapeutics-Minireview Absorption, Distribution, Metabolism, and Excretion of US Food and Drug Administration-Approved Antisense Oligonucleotide Drugs [J].
Migliorati, Julia M. ;
Liu, Sunna ;
Liu, Anna ;
Gogate, Anagha ;
Nair, Sreenidhi ;
Bahal, Raman ;
Rasmussen, Theodore P. ;
Manautou, Jose E. ;
Zhong, Xiao-bo .
DRUG METABOLISM AND DISPOSITION, 2022, 50 (06) :888-897
[5]   Phase 1-2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS [J].
Miller, Timothy ;
Cudkowicz, Merit ;
Shaw, Pamela J. ;
Andersen, Peter M. ;
Atassi, Nazem ;
Bucelli, Robert C. ;
Genge, Angela ;
Glass, Jonathan ;
Ladha, Shafeeq ;
Ludolph, Albert L. ;
Maragakis, Nicholas J. ;
McDermott, Christopher J. ;
Pestronk, Alan ;
Ravits, John ;
Salachas, Francois ;
Trudell, Randall ;
Van Damme, Philip ;
Zinman, Lorne ;
Bennett, C. Frank ;
Lane, Roger ;
Sandrock, Alfred ;
Runz, Heiko ;
Graham, Danielle ;
Houshyar, Hani ;
McCampbell, Alexander ;
Nestorov, Ivan ;
Chang, Ih ;
McNeill, Manjit ;
Fanning, Laura ;
Fradette, Stephanie ;
Ferguson, Toby A. .
NEW ENGLAND JOURNAL OF MEDICINE, 2020, 383 (02) :109-119
[6]   Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS [J].
Miller, Timothy M. ;
Cudkowicz, Merit E. ;
Genge, Angela ;
Shaw, Pamela J. ;
Sobue, Gen ;
Bucelli, Robert C. ;
Chio, Adriano ;
Van Damme, Philip ;
Ludolph, Albert C. ;
Glass, Jonathan D. ;
Andrews, Jinsy A. ;
Babu, Suma ;
Benatar, Michael ;
McDermott, Christopher J. ;
Cochrane, Thos ;
Chary, Sowmya ;
Chew, Sheena ;
Zhu, Han ;
Wu, Fan ;
Nestorov, Ivan ;
Graham, Danielle ;
Sun, Peng ;
McNeill, Manjit ;
Fanning, Laura ;
Ferguson, Toby A. ;
Fradette, Stephanie .
NEW ENGLAND JOURNAL OF MEDICINE, 2022, 387 (12) :1099-1110
[7]   A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration [J].
Monine, Michael ;
Norris, Daniel ;
Wang, Yanfeng ;
Nestorov, Ivan .
JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS, 2021, 48 (05) :639-654
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