What question did this study set out to answer?

Provide expert consensus recommendations for selecting therapies and monitoring disease progression and treatment response in hereditary and wild-type ATTR amyloidosis.

June 2, 2022Amyloid

Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis

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Authors

DADavid AdamsInterventional / Structural Cardiology MBMerrill D. BensonSouthwestern Medical Center JBJohn L. BerkBoston University

Discussion

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Implication

Expert consensus establishes clinical guidelines for therapy and monitoring in ATTR amyloidosis, highlighting the shifting roles of gene silencers and kinetic stabilizers.

Key Points

Provide expert consensus recommendations for selecting therapies and monitoring disease progression and treatment response in hereditary and wild-type ATTR amyloidosis.
Synthesized evidence from phase III clinical trials evaluating gene silencers and kinetic stabilizers targeting the transthyretin amyloid cascade.
Formulated consensus recommendations for therapy selection, monitoring strategies, and evaluating disease progression across neuropathic and cardiac presentations.
Targeted gene silencers and kinetic stabilizers demonstrate clinical efficacy in slowing amyloid neuropathy and cardiomyopathy, significantly narrowing the indications for liver transplantation.
Absence of direct head-to-head comparative clinical trials limits definitive hierarchy among approved disease-modifying agents, necessitating individualized monitoring protocols.

Cite This Study

Adams et al. (2022) studied this question.

synapsesocial.com/papers/6aaa37e724cd5fa6fa547653 https://doi.org/10.1080/13506129.2022.2052838

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Implication

Expert consensus establishes clinical guidelines for therapy and monitoring in ATTR amyloidosis, highlighting the shifting roles of gene silencers and kinetic stabilizers.

Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis

Discussion

Member takes

Implication

Key Points

Cite This Study

Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis

Discussion

Member takes

Implication

Key Points

Cite This Study

Also Consider

Also Consider