Antisense oligonucleotides
Neurology· 3 reviewed drugs· 3758 cited findings behind them
What the regulatory reviews cover
Antisense oligonucleotides (ASOs) in this library include Qalsody, Wainzua, and TEGSEDI. These drugs have been reviewed for various indications, including the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (Qalsody), and hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy (Wainzua and TEGSEDI).
Regulatory review documents report different types of results for these ASOs. For Qalsody, the median disease onset in SOD1-G93A mice (tofersen) was 168 days, compared to 147.5 days for control ASO. Wainzua demonstrated hTTR mRNA expression inhibition potency (eplontersen vs inotersen) as 51-fold more potent. TEGSEDI showed a Treatment Difference (LS Mean) in mNIS+7 of -19.7. It is important to note that these figures are derived from different trials and are not head-to-head comparisons between the drugs.
The drugs — one cited result each
| Qalsody Biogen Inc. | Median disease onset in SOD1-G93A mice (tofersen) at 30 days of age: 168 Biogen B V — Qalsody EMA EPAR public assessment report (EMEA, p.23 |
| Wainzua AstraZeneca PLC | hTTR mRNA expression inhibition potency (eplontersen vs inotersen): 51-fold more potent AstraZeneca PLC — Wainzua EMA EPAR public assessment report, p.26 |
| TEGSEDI Ionis Pharmaceuticals, Inc. | Treatment Difference (LS Mean) in mNIS+7: -19.7 AKCEA THERAPS — Tegsedi FDA Review (NDA/BLA 211172, 2026), p.29 |
Different drugs were studied in different trials — these figures are not head-to-head comparisons.
Explore the full evidence
Every finding behind these drugs — searchable, comparable and answerable in plain language — is in the Neurology research library →
Lists reflect the regulatory review documents in this library, not all therapies that exist. Educational reference only — not medical or investment advice.