Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study

@article{PlantBordeneuve2016LongtermTO,
  title={Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study},
  author={Violaine Plant{\'e}-Bordeneuve and Farida Gorram and Hayet Salhi and Tarik Nordine and Samar S Ayache and Philippe Le Corvoisier and Daniel Azoulay and Cyrille F{\'e}ray and Thibaud Damy and J-P Lefaucheur},
  journal={Journal of Neurology},
  year={2016},
  volume={264},
  pages={268-276}
}
Tafamidis is a transthyretin (TTR) stabilizer recently approved to slow the neurologic impairment in TTR familial amyloid polyneuropathy (TTR-FAP). The pivotal studies on Tafamidis reported encouraging results on the short term, in the early onset Val30Met-TTR-FAP patients at an early stage of the neuropathy. However, the effect of the drug in the non-Val30Met patients, at a more advanced stage of the disease and on the long term, is less known. In this study, we report the effect of Tafamidis… CONTINUE READING
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