Literature DB >> 34783948

Targeted treatments of AL and ATTR amyloidosis.

Pranav Chandrashekar1, Anish K Desai2, Barry H Trachtenberg3,4.   

Abstract

The therapeutic landscape for cardiac amyloidosis is rapidly evolving. In the last decade, our focus has shifted from dealing with the inevitable complications of continued extracellular infiltration of amyloid fibrils to earlier identification of these patients with prompt initiation of targeted therapy to prevent further deposition. Although much of the focus on novel targeted therapies is within the realm of transthyretin amyloidosis, light chain amyloidosis has benefited due to an overlap particularly in the final common pathway of fibrillogenesis and extraction of amyloid fibrils from the heart. Here, we review the targeted therapeutics for transthyretin and light chain amyloidosis. For transthyretin amyloidosis, the list of current and future therapeutics continues to evolve; and therefore, it is crucial to become familiar with the underlying mechanistic pathways of the disease. Although targeted therapeutic choices in AL amyloidosis are largely driven by the hematology team, the cardiac adverse effect profiles of these therapies, particularly in those with advanced amyloidosis, provide an opportunity for early recognition to prevent decompensation and can help inform recommendations regarding therapy changes when required.
© 2021. The Author(s), under exclusive licence to Springer Science+Business Media, LLC, part of Springer Nature.

Entities:  

Keywords:  Amyloidosis; Cardiomyopathy; Light chain; Targeted therapeutics; Transthyretin

Mesh:

Substances:

Year:  2021        PMID: 34783948     DOI: 10.1007/s10741-021-10180-z

Source DB:  PubMed          Journal:  Heart Fail Rev        ISSN: 1382-4147            Impact factor:   4.654


  105 in total

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5.  A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing.

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Journal:  Sci Rep       Date:  2017-11-15       Impact factor: 4.379

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Journal:  Circulation       Date:  2016-04-22       Impact factor: 29.690

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  1 in total

Review 1.  Future Directions in Cardiac Amyloidosis.

Authors:  Barry Trachtenberg
Journal:  Methodist Debakey Cardiovasc J       Date:  2022-03-14
  1 in total

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