Category «Duchenne Muscular Dystrophy»

Golodirsen, ゴロジルセン;

It’s only fair to share…   Golodirsen RNA, [P-deoxy-P-(dimethylamino)](2′,3′-dideoxy-2′,3′-imino-2′,3′-seco)(2’a→5′)(G-m5U-m5U-G-C-C-m5U-C-C-G-G-m5U-m5U-C-m5U-G-A-A-G-G-m5U-G-m5U-m5U-C), 5′-[P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylphosphonamidate] Nucleic Acid Sequence Sequence Length: 25 Formula C305H481N138O112P25 CAS 1422959-91-8 Mol weight 8647.2841   Exon 53: NG-12-0163 Golodirsen SRP 4053 Nucleic Acid Sequence Sequence Length: 252 a 6 c 8 g 9 umodified FDA APPROVED, Vyondys 53, 019/12/12 Antisense oligonucleotide ゴロジルセン; Duchenne muscular dystrophy (DMD variant amenable …