Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis | oneAMYLOIDOSISvoice
×
Or register here
Global Search

Trusted Resources: Education

Scientific literature and patient education texts

Back to Education / Scientific Articles

Emerging Therapeutics for the Treatment of Light Chain and Transthyretin Amyloidosis

key information

source: JACC. Basic to Translational Science

year: 2019

authors: Zhang KW, Stockerl-Goldstein KE, Lenihan DJ

summary/abstract:

Cardiac amyloidosis is a restrictive cardiomyopathy that results from the deposition of misfolded light chain or transthyretin proteins, most commonly, in cardiac tissue. Traditionally, treatment options for light chain (AL) and transthyretin (ATTR) amyloidosis have been limited. However, there are now multiple novel therapeutics in development and several therapeutics recently approved that promise to revolutionize clinical management of AL and ATTR. Most of these agents disrupt specific stages of amyloidogenesis such as light chain or transthyretin protein production, formation of amyloidogenic intermediates, or amyloid fibril aggregation. Others aim to remove existing amyloid tissue deposits using monoclonal antibody technology. Although these advances represent an important step forward in the care of cardiac amyloidosis patients, additional studies are needed to define the optimal treatment paradigms for AL and ATTR and to validate clinical, imaging, or serum biomarker strategies that may confirm a cardiac response to therapy.

organization: Washington University in St. Louis School of Medicine, USA

DOI: 10.1016/j.jacbts.2019.02.002

read more full text

Related Content

To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences.
More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.

Close

Community Key Collaborator

Support for this landing page is provided by an independent education grant from

Translation of this platform made possible through an independent education grant from

© 2025 rareLife solutions Inc.

To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences.
More information

The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.

Close