1. Ionis Pharmaceuticals [Internet]. Annual Report, 2022 [last accessed 11 December 2023]. Available from: https://ir.ionispharma.com/static-files/db9dff5d-8683-485a-a517-15e264fe7532.
2. Coelho T, et al. Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-LRx(ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy. Nerol Ther. 2021 Jun;10(1):375-389.
3. Lin X, et al. BMC Neurol. 2021 Feb 12;21(1):70.
4. Adams D, et al. J Neurol. 2021 Jun;268(6):2109-2122.
5. Gillmore JD et al. Cardiol Ther. 2025. DOI: 10.1007/s40119-025-00402-y.
6. Coelho T, et al. Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy. JAMA. 2023;330(15):1448–1458.
7. Coelho T, et al. Characteristics of patients with hereditary transthyretin amyloidosis-polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an open-label phase 3 study of eplontersen. Neurol Ther. 2023;12(1):267-287.
8. Ionis Pharmaceuticals [Internet]. 2023 Annual Report [cited 2024 September 13]. Available from: https://ir.ionis.com/static-files/aad58173-0ab5-4142-b6d4-3e940c7eb2a6.
9. AstraZeneca [Internet]. Press release. Wainua (eplontersen) granted first-ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis [cited 2024 October 8]. Available from: https://www.astrazeneca.com/media-centre/press-releases/2023/Wainua-eplontersen-granted-first-ever-regulatory-approval-us-treatment-of-adults-with-polyneuropathy-hereditary-transthyretin-mediated-amyloidosis.html.
10. ClinicalTrials.gov [Internet]. CARDIO-TTRansform: a study to evaluate the efficacy and safety of eplontersen (formerly known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in participants with transthyretin-mediated amyloid cardiomyopathy (ATTR CM) [cited 2024 October 8]. Available from https://www.clinicaltrials.gov/study/NCT04136171.
11. Ionis [Internet]. Ionis completes enrollment in landmark Phase 3 CARDIO-TTRansform study in patients with TTR-mediated amyloid cardiomyopathy. 2023 July 31 [cited 2024 October 8]. Available from https://ir.ionis.com/news-releases/news-release-details/ionis-completes-enrollment-landmark-phase-3-cardio-ttransform#:~:text=CARDIO%2DTTRansform%20is%20the%20largest,threatening%20cardiovascular%20(CV)%20events.
12. Rintell D, et al. Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups. Orphanet J Rare Dis. 2021;16:70.
