カシメルセン;
Casimersen (USAN/INN)

C268H424N124O95P22

7580.6386

7584.44

RNA, [P-deoxy-P-(dimethylamino)
](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco) (2′a→5′)
(C-A-A-m5U-G-C-C-A-m5U-C-C-m5U-G-G-A-G-m5U-m5U-C-C-m5U-G) ,
5′-[P-[4-[[2-[2-(2-hydroxyethoxy)
ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate]

翻訳阻害薬

DMD* [HSA_VAR:1756v1] [HSA:1756] [KO:K10366] (pre-mRNA exon45)