Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy

Tafamidis, a transthyretin (TTR) kinetic stabilizer, delayed neuropathic progression in patients with Val30Met TTR familial amyloid polyneuropathy (TTR-FAP) in an 18-month randomized controlled trial (study Fx-005). This 12-month, open-label extension study evaluated the long-term safety, tolerabili...

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Autori principali: Coelho, Teresa, Maia, Luis F., da Silva, Ana Martins, Cruz, Márcia W., Planté-Bordeneuve, Violaine, Suhr, Ole B., Conceiçao, Isabel, Schmidt, Hartmut H.-J., Trigo, Pedro, Kelly, Jeffery W., Labaudinière, Richard, Chan, Jason, Packman, Jeff, Grogan, Donna R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer Berlin Heidelberg 2013
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Original Communication
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3825212/
https://ncbi.nlm.nih.gov/pubmed/23974642
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-013-7051-7
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