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Wirkstoff(e)
- 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole
- Adeno-associated viral vector serotype 9 containing the human SMN gene
- Phosphorothioate-Oligonukleotid spezifisch für Exon 51
- RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a-->5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl] N,N-dimethylaminophosphonamidate