Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS
2020
Abstract Background Tofersen is an antisense oligonucleotide that mediates the degradation of superoxide dismutase 1 (SOD1) messenger RNA to reduce SOD1 protein synthesis. Intrathecal administratio...
Keywords:
- Correction
- Source
- Cite
- Save
- Machine Reading By IdeaReader
18
References
95
Citations
NaN
KQI