SPINRAZA® contains the active substance nusinersen, which belongs to a group of medicines known as antisense oligonucleotides (ASOs).1
SPINRAZA® increases the proportion of SMN2 mRNA transcripts that include exon 7 by targeting SMN2 pre-messenger ribonucleic acid (pre-mRNA).1
Once SMN2 mRNA with exon 7 is produced, it can be translated into functional, full-length SMN protein.1
age 9*Later-onset (Type III) SMA
treated with SPINRAZA®
~5.5 years as of 9/18
1. SPINRAZA® Summary of Product Characteristics.