Effects of Tafamidis on Transthyretin Stabilization and Clinical Outcomes in Patients with Non-Val30Met Transthyretin Amyloidosis

This phase II, open-label, single-treatment arm study evaluated the pharmacodynamics, efficacy, and safety of tafamidis in patients with non-Val30Met transthyretin (TTR) amyloidosis. Twenty-one patients with eight different non-Val30Met mutations received 20 mg QD of tafamidis meglumine for 12 month...

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Autores principales: Merlini, Giampaolo, Planté-Bordeneuve, Violaine, Judge, Daniel P., Schmidt, Hartmut, Obici, Laura, Perlini, Stefano, Packman, Jeff, Tripp, Tara, Grogan, Donna R.
Formato: Artigo
Lenguaje:Inglês
Publicado: Springer US 2013
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3838581/
https://ncbi.nlm.nih.gov/pubmed/24101373
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s12265-013-9512-x
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