| Nusinersen |
Spinal Muscular Atrophy (SMA) |
ASO |
Splicing switch exon 7 inclusion |
2016 |
| Macugen |
Neovascular age-related Macular Dgeneration (AMD) |
Aptamer |
Steric block |
2004 |
| Fomivirsen |
Cytomegalovirus Rhinitis |
ASO |
Steric block |
1998 |
| Mipomersen |
Familial hypercholesterolemia (FH) |
ASO |
RNase H-mediated mRNA degradation |
2013 |
| Eteplirsen |
Duchenne muscular dystrophy |
ASO |
Splicing switch exon 51 Skipping |
2016 |
| Inotersen |
Hereditary transthyretin amyloidosis |
ASO |
RNase H-mediated mRNA degradation |
2018 |
| Patisiran |
Hereditary TTR-mediated amyloidosis (hATTR) |
siRNA |
Ago 2 |
2018 |
| Volanesorsen |
Familial Chylomicronemia |
ASO |
RNase H-mediated mRNA degradation |
2019 |
| Givosiran |
Acute hepatic porphyria (AHP) |
siRNA |
Ago 2 |
2019 |
| Golodirsen |
Duchenne muscular dystrophy (DMD) |
ASO |
Splicing switch exon 53 Skipping |
2019 |
| Lumasiran |
Primary Hyperoxaluria type 1 (PH1) |
siRNA |
Reduce glycolate oxidase (GO) enzyme |
2020 |
