Nusinersen is an antisense oligonucleotide that induces SMN protein expression. Nusinersen was developed in 2016 for the treatment of spinal muscular atrophy (SMA), a rare neuromuscular disorder.
Synonyms:
RNA [2'-O-(2-methoxyethyl)](P-thio)(m5U-m5C-A-m5C-m5U-m5U-m5U-m5C-A-m5U-A-A-m5U-G-m5C-m5U-G-G);
Spinraza;
ISIS 396443;
RNA, [2'-O-(2-methoxyethyl)](P-thio)(m5U-m5C-A-m5C-m5U-m5U-m5U-m5C-A-m5U-A-A-m5U-G-m5C-m5U-G-G);
1: PN: WO2010148249 SEQID: 1 claimed DNA;