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Real-world treatment management in hereditary transthyretin amyloidosis – an experience report and proposal for therapy switch decision criteria

Abstract Background Hereditary transthyretin amyloidosis is a rapidly progressive and lethal disease. Thanks to the increasing number of disease-modifying treatments, prognosis has improved significantly. However, new challenges regarding treatment response and when to change treatment remain unansw...

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Principais autores: Duc Chu Dieu, Helena F. Pernice, Harisa Muratovic, Paul J. Wetzel, Gina Barzen, Nicolas W. Wieder, Stefanie M. Werhahn, Bettina Heidecker, Sebastian Spethmann, Katrin Hahn
Format: Artigo
Sprog:Inglês
Udgivet: BMC 2025-09-01
Serier:Neurological Research and Practice
Fag:
Hereditary transthyretin amyloidosis
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Vutrisiran
Tafamidis
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SiRNA
Online adgang:https://doi.org/10.1186/s42466-025-00428-6
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