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Evaluation of tafamidis as first-line therapeutic agent for transthyretin familial amyloidotic polyneuropathy

Almost 100 mutations in the human transthyretin (TTR) gene cause the autosomal dominant disorders of familial amyloidotic polyneuropathy (FAP) and familial amyloidotic cardiomyopathy. While these have been clinically classified as separate disorders, the peripheral and autonomic nervous systems and...

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Dettagli Bibliografici
Pubblicato in:Degener Neurol Neuromuscul Dis
Autore principale: Buxbaum, Joel N
Natura: Artigo
Lingua:Inglês
Pubblicazione: Dove Medical Press 2012
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6065585/
https://ncbi.nlm.nih.gov/pubmed/30890886
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.2147/DNND.S24624
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