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Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial

OBJECTIVES: To evaluate the efficacy and safety of 18 months of tafamidis treatment in patients with early-stage V30M transthyretin familial amyloid polyneuropathy (TTR-FAP). METHODS: In this randomized, double-blind trial, patients received tafamidis 20 mg QD or placebo. Coprimary endpoints were th...

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Detalhes bibliográficos
Main Authors: Coelho, Teresa, Maia, Luis F., Martins da Silva, Ana, Waddington Cruz, Marcia, Planté-Bordeneuve, Violaine, Lozeron, Pierre, Suhr, Ole B., Campistol, Josep M., Conceição, Isabel Maria, Schmidt, Hartmut H.-J., Trigo, Pedro, Kelly, Jeffery W., Labaudinière, Richard, Chan, Jason, Packman, Jeff, Wilson, Amy, Grogan, Donna R.
Formato: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2012
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4098875/
https://ncbi.nlm.nih.gov/pubmed/22843282
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0b013e3182661eb1
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