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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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Bibliografiske detaljer
Udgivet i:Degener Neurol Neuromuscul Dis
Hovedforfatter: Buxbaum, Joel N
Format: Artigo
Sprog:Inglês
Udgivet: Dove Medical Press 2012
Fag:
Online adgang: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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