inotersen (Tegsedi)
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Indications
- treatment of hereditary transthyretin amyloidosis
- treatment of associated polyneuropathy[3] dosage:
- 300 mg sq weekly
Adverse effects
- glomerulonephritis (3%)*
- thrombocytopenia (3%)*
- mortality (4%)
Laboratory
Mechanism of action
- inhibits hepatic synthesis of transthyretin
More general terms
- short interfering double-stranded RNA; antisense oligonucleotide (siRNA, microRNA, miRNA)
- metabolic agent (metabolic modifier)
References
- ↑ Benson MD, Waddington-Cruz M, Berk JL et al Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis N Engl J Med 2018; 379:22-31. July 5. PMID: https://www.ncbi.nlm.nih.gov/pubmed/29972757 https://www.nejm.org/doi/full/10.1056/NEJMoa1716793
- ↑ Yasgur BS New Agents Show Promise for Severe and Fatal Genetic Disease. Medscape - Jul 10, 2018. https://www.medscape.com/viewarticle/899112
- ↑ 3.0 3.1 George J FDA Greenlights Inotersen for Rare Disease Nerve Pain - But approval comes with boxed warning and mandatory monitoring. MedPage Today, October 06, 2018 https://www.medpagetoday.com/neurology/generalneurology/75546
- ↑ RxNorm
- ↑ Brooks M FDA OKs Inotersen (Tegsedi) for Hereditary ATTR With Polyneuropathy. Medscape - Oct 08, 2018. https://www.medscape.com/viewarticle/903104