Products Nusinersen was approved in the United States in 2016 and in many countries and the EU in 2017 as a solution for intrathecal injection by lumbar puncture and as the first drug for SMA treatment (Spinraza). Structure and properties Nusinersen is a modified antisense oligonucleotide. Effects Nusinersen (ATC M09AX07) increases the formation of the … Nusinersen
Products Eteplirsen (Sarepta Therapeutics Inc) was approved in the United States in 2016 (Exondys 51). Structure and properties Eteplirsen (C364H569N177O122P30, Mr = 10305.7 g/mol) is a phosphorodiamidate morpholino oligomer (PMO) that binds selectively to exon 51 of pre-mRNA. It is an antisense oligomer with a high molecular weight. The sequence is complementary to the segment … Eteplirsen
