Tegsedi (Inotersen): An antisense oligonucleotide approved for the treatment of adult patients with hereditary transthyretin amyloidosis
Tegsedi (Inotersen) is a chemicallymodified antisense oligonucleotide that inhibits the hepatic production of transthyretin (TTR). Several single-point mutations in TTR destabilize its structure, leading to the aggregation and accumulation of amyloid deposits in the nervous system, heart, kidneys an...
| Autor principal: | |
|---|---|
| Formato: | article |
| Idioma: | eng |
| Publicado em: |
2019
|
| Assuntos: | |
| Texto completo: | https://hdl.handle.net/10216/136219 |
| País: | Portugal |
| Oai: | oai:repositorio-aberto.up.pt:10216/136219 |