Fig. 3From: The potential of antisense oligonucleotide therapies for inherited childhood lung diseasesSPINRAZA® strengthens exon 7 recognition and retention in the SMN2 mRNA transcript, reducing SMA disease severity. a Predominant splicing of SMN2 exons 6–9. b An exonic polymorphism weakens exon 7 selection in the SMN2 mRNA. SPINRAZA® strengthens exon 7 selection in the SMN2 transcript, producing a functional protein and reducing SMA disease severityBack to article page