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Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy.

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Title: Rationale and Design of CARDIO-TTRansform, a Phase 3 Trial of Eplontersen in Transthyretin Amyloid Cardiomyopathy.
Authors: Masri A; Division of Cardiovascular Medicine, Oregon Health and Sciences University, Portland (A.M.)., Cappelli F; Tuscan Regional Amyloidosis Center, Department of Clinical and Experimental Medicine, University Hospital Careggi, Florence, Italy (F.C.)., Davis MK; Division of Cardiology, University of British Columbia, Vancouver, Canada (M.K.D.)., Fontana M; National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, United Kingdom (M.F., J.D.G.)., Garcia-Pavia P; Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV and Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain (P.G.-P.)., Gillmore JD; National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, United Kingdom (M.F., J.D.G.)., Hanna M; Department of Cardiovascular Medicine, Cleveland Clinic, OH (M.H.)., Obici L; Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy (L.O.)., Solomon SD; Cardiovascular Division, Brigham and Women's Hospital, Boston, MA (S.D.S.)., Sperry BW; Saint Luke's Mid America Heart Institute, Kansas City, MO (B.W.S.)., Tahara N; Division of Cardiovascular Medicine, Department of Medicine, Kurume University School of Medicine, Japan (N.T.)., Waddington-Cruz M; Centro de Paramiloidose Antônio Rodrigues de Mello (CEPARM), Amyloidosis Center, University Hospital, Federal University of Rio de Janeiro, Brazil (M.W.-C.).; Amyloidosis Center, Complexo Hospitalar Américas-Samaritano-Vitória, Rio de Janeiro, Brazil (M.W.-C.)., Kristen AV; Amyloidosis Center, University of Heidelberg, Germany (A.V.K.).; Cardiovascular Center Darmstadt, Germany (A.V.K.)., Falk RH; Harvard Medical School and Brigham and Women's Hospital, Boston, MA (R.H.F.)., Shah SJ; Northwestern University Feinberg School of Medicine, Chicago, IL (S.J.S.)., Nativi-Nicolau J; Mayo Clinic, Jacksonville, FL (J.N.-N.)., Viney NJ; Ionis Pharmaceuticals, Inc., Carlsbad, CA (N.J.V., Q.Y., S.T.)., Yang Q; Ionis Pharmaceuticals, Inc., Carlsbad, CA (N.J.V., Q.Y., S.T.)., Chen J; Late-Stage Clinical Development, Cardiovascular, Renal and Metabolism (CVRM), BioPharmaceuticals R&D, AstraZeneca, Gaithersburg, MA (J.C.)., Tsimikas S; Ionis Pharmaceuticals, Inc., Carlsbad, CA (N.J.V., Q.Y., S.T.)., Maurer MS; Columbia University Irving Medical Center, New York, NY (M.S.M.).
Source: Circulation. Heart failure [Circ Heart Fail] 2026 Jun; Vol. 19 (6), pp. e014205. Date of Electronic Publication: 2026 May 09.
Publication Type: Journal Article; Randomized Controlled Trial; Clinical Trial, Phase III
Journal Info: Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 101479941 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1941-3297 (Electronic) Linking ISSN: 19413289 NLM ISO Abbreviation: Circ Heart Fail Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Masri+A%22">Masri A</searchLink>; Division of Cardiovascular Medicine, Oregon Health and Sciences University, Portland (A.M.).<br /><searchLink fieldCode="AU" term="%22Cappelli+F%22">Cappelli F</searchLink>; Tuscan Regional Amyloidosis Center, Department of Clinical and Experimental Medicine, University Hospital Careggi, Florence, Italy (F.C.).<br /><searchLink fieldCode="AU" term="%22Davis+MK%22">Davis MK</searchLink>; Division of Cardiology, University of British Columbia, Vancouver, Canada (M.K.D.).<br /><searchLink fieldCode="AU" term="%22Fontana+M%22">Fontana M</searchLink>; National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, United Kingdom (M.F., J.D.G.).<br /><searchLink fieldCode="AU" term="%22Garcia-Pavia+P%22">Garcia-Pavia P</searchLink>; Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, IDIPHISA, CIBERCV and Centro Nacional de Investigaciones Cardiovasculares (CNIC), Madrid, Spain (P.G.-P.).<br /><searchLink fieldCode="AU" term="%22Gillmore+JD%22">Gillmore JD</searchLink>; National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital, United Kingdom (M.F., J.D.G.).<br /><searchLink fieldCode="AU" term="%22Hanna+M%22">Hanna M</searchLink>; Department of Cardiovascular Medicine, Cleveland Clinic, OH (M.H.).<br /><searchLink fieldCode="AU" term="%22Obici+L%22">Obici L</searchLink>; Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy (L.O.).<br /><searchLink fieldCode="AU" term="%22Solomon+SD%22">Solomon SD</searchLink>; Cardiovascular Division, Brigham and Women's Hospital, Boston, MA (S.D.S.).<br /><searchLink fieldCode="AU" term="%22Sperry+BW%22">Sperry BW</searchLink>; Saint Luke's Mid America Heart Institute, Kansas City, MO (B.W.S.).<br /><searchLink fieldCode="AU" term="%22Tahara+N%22">Tahara N</searchLink>; Division of Cardiovascular Medicine, Department of Medicine, Kurume University School of Medicine, Japan (N.T.).<br /><searchLink fieldCode="AU" term="%22Waddington-Cruz+M%22">Waddington-Cruz M</searchLink>; Centro de Paramiloidose Antônio Rodrigues de Mello (CEPARM), Amyloidosis Center, University Hospital, Federal University of Rio de Janeiro, Brazil (M.W.-C.).; Amyloidosis Center, Complexo Hospitalar Américas-Samaritano-Vitória, Rio de Janeiro, Brazil (M.W.-C.).<br /><searchLink fieldCode="AU" term="%22Kristen+AV%22">Kristen AV</searchLink>; Amyloidosis Center, University of Heidelberg, Germany (A.V.K.).; Cardiovascular Center Darmstadt, Germany (A.V.K.).<br /><searchLink fieldCode="AU" term="%22Falk+RH%22">Falk RH</searchLink>; Harvard Medical School and Brigham and Women's Hospital, Boston, MA (R.H.F.).<br /><searchLink fieldCode="AU" term="%22Shah+SJ%22">Shah SJ</searchLink>; Northwestern University Feinberg School of Medicine, Chicago, IL (S.J.S.).<br /><searchLink fieldCode="AU" term="%22Nativi-Nicolau+J%22">Nativi-Nicolau J</searchLink>; Mayo Clinic, Jacksonville, FL (J.N.-N.).<br /><searchLink fieldCode="AU" term="%22Viney+NJ%22">Viney NJ</searchLink>; Ionis Pharmaceuticals, Inc., Carlsbad, CA (N.J.V., Q.Y., S.T.).<br /><searchLink fieldCode="AU" term="%22Yang+Q%22">Yang Q</searchLink>; Ionis Pharmaceuticals, Inc., Carlsbad, CA (N.J.V., Q.Y., S.T.).<br /><searchLink fieldCode="AU" term="%22Chen+J%22">Chen J</searchLink>; Late-Stage Clinical Development, Cardiovascular, Renal and Metabolism (CVRM), BioPharmaceuticals R&D, AstraZeneca, Gaithersburg, MA (J.C.).<br /><searchLink fieldCode="AU" term="%22Tsimikas+S%22">Tsimikas S</searchLink>; Ionis Pharmaceuticals, Inc., Carlsbad, CA (N.J.V., Q.Y., S.T.).<br /><searchLink fieldCode="AU" term="%22Maurer+MS%22">Maurer MS</searchLink>; Columbia University Irving Medical Center, New York, NY (M.S.M.).
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  Data: <searchLink fieldCode="JN" term="%22101479941%22">Circulation. Heart failure</searchLink> [Circ Heart Fail] 2026 Jun; Vol. 19 (6), pp. e014205. <i>Date of Electronic Publication: </i>2026 May 09.
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Lippincott+Williams+%26+Wilkins%22">Lippincott Williams & Wilkins </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101479941 <i>Publication Model: </i>Print-Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>1941-3297 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2219413289%22">19413289 </searchLink><i>NLM ISO Abbreviation: </i>Circ Heart Fail <i>Subsets: </i>MEDLINE
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              Text: 2026 Jun
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