Tafamidis for transthyretin familial amyloid polyneuropathy
Fundación para la Lucha contra las Enfermedades Neurológicas de la Infancia · Hospital Universitário Clementino Fraga Filho · +3 more institutions
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Abstract
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 the Neuropathy Impairment Score-Lower Limbs (NIS-LL) responder analysis (
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792
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Authors
24Topics & keywords
Topics
Keywords
- Medicine
- Internal medicine
- Placebo
- Population
- Clinical endpoint
- Adverse effect
- Polyneuropathy
- Transthyretin
UN Sustainable Development Goals
- Zero hunger
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