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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Autori principali:	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
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	BMC 2025-09-01
Serie:	Neurological Research and Practice
Soggetti:	Hereditary transthyretin amyloidosis Patisiran Vutrisiran Tafamidis Inotersen SiRNA
Accesso online:	https://doi.org/10.1186/s42466-025-00428-6
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