ArticlePharmaceuticals (Basel, Switzerland)2019
Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis.
Article in Pharmaceuticals (Basel, Switzerland), 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 41 papers.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
41 citing papers in PubMed, 73 citations in OpenAlex.
- Multi-Omics-Guided Design and Safety Engineering of Nucleic Acid Therapeutics: From Molecular Perturbation to Predictive Toxicology and Precision Translation.Chemical biology & drug design · 2026Review
- Eplontersen for the Treatment of Hereditary Transthyretin Amyloidosis.Methods in molecular biology (Clifton, N.J.) · 2026Article
- Target, silence, replace: a review on RNA-based drugs in modern medicine.Frontiers in cell and developmental biology · 2026Review
- Comparative Study on Cardiac Findings in Patients with Transthyretin Amyloidosis Before and After Treatment with a Transthyretin Silencer.Journal of cardiovascular development and disease · 2025Article
- RNA therapeutics in kidney diseases: prospects and current status.Clinical kidney journal · 2025Review
- Endogenous Ribonucleases: Therapeutic Targeting of the Transcriptome Through Oligonucleotide-Triggered RNA Inactivation.Biomolecules · 2025Review
- Pharmaceutical perspectives on oligonucleotide therapeutics and delivery systems.Pharmacological reviews · 2025Review
- RNA Therapeutics: Focus on Antisense Oligonucleotides in the Nervous System.Biomolecules & therapeutics · 2025Review
- Emerging, novel gene-modulating therapies for transthyretin amyloid cardiomyopathy.Heart failure reviews · 2025Review
- RNA therapeutics in cardiovascular medicine.Current opinion in cardiology · 2025Review
- Chemical engineering of CRISPR-Cas systems for therapeutic application.Nature reviews. Drug discovery · 2025Review
- Review
- Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington's disease.Translational neurodegeneration · 2025Review
- X-inactive specific transcript (XIST) can determine sex differences in cardiovascular drug responses: focus on RNA therapeutics.Frontiers in pharmacology · 2025Article
- Nucleic acid drugs: recent progress and future perspectives.Signal transduction and targeted therapy · 2024Review
- Amphiphilic Oligonucleotide Derivatives-Promising Tools for Therapeutics.Pharmaceutics · 2024Review
- Strategies to improve the design of gapmer antisense oligonucleotide on allele-specific silencing.Molecular therapy. Nucleic acids · 2024Article
- Review
- Mechanisms of Action of the US Food and Drug Administration-Approved Antisense Oligonucleotide Drugs.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- Intrinsic and Extrinsic Contributors to the Cardiac Benefits of Exercise.JACC. Basic to translational science · 2024Review
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
Tegsedi (Inotersen) is a chemically modified 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 and eyes. In July 2018, Tegsedi was approved by the European Commission for use in adults with stage one and two polyneuropathies. Later on, in October 2018, the FDA and Health Canada also approved its use for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults in the U.S. and Canada. Tegsedi was developed by Ionis Pharmaceuticals, the company that holds the global marketing license, together with its subsidiary Akcea Therapeutics.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.