Evolving landscape in the management of transthyretin amyloidosis.

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Published in Ann Med on November 27, 2015

Authors

Philip N Hawkins1, Yukio Ando2, Angela Dispenzeri3, Alejandra Gonzalez-Duarte4, David Adams5, Ole B Suhr6

Author Affiliations

1: a National Amyloidosis Centre, Royal Free Hospital, University College London , London , UK.
2: b Department of Neurology , Graduate School of Medical Sciences Kumamoto University , Kumamoto , Japan.
3: c Mayo Clinic, Division of Hematology , Rochester, Minnesota, USA.
4: d Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán , Mexico City , Mexico.
5: e National Reference Centre for FAP, APHP, CHU Bicêtre, Université Paris-Sud, INSERM U788 , Paris , France.
6: f Department of Public Health and Clinical Medicine , Umeå University , Umeå , Sweden.

Associated clinical trials:

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis | NCT01960348

Safety and Efficacy of Tafamidis in Patients With Transthyretin Cardiomyopathy (ATTR-ACT) | NCT01994889

The Study of an Investigational Drug, ALN-TTR02 (Patisiran), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran) | NCT01961921

Efficacy and Safety of IONIS-TTR Rx in Familial Amyloid Polyneuropathy | NCT01737398

Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis | NCT01981837

A Study to Evaluate the Safety of GSK2398852 When Co-administered With GSK2315698 in Patients With Systemic Amyloidosis | NCT01777243

Safety, Efficacy and Pharmacokinetics of Doxycycline Plus Tauroursodeoxycholic Acid in Transthyretin Amyloidosis | NCT01171859

Tolerability and Efficacy of a Combination of Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy | NCT01855360

Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis | NCT01707264

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | NCT02319005

Open-Label Extension Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP) | NCT02175004

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