Early data on long‐term efficacy and safety of inotersen in patients with hereditary transthyretin amyloidosis: a 2‐year update from the open‐label extension of the NEURO‐TTR trial

BACKGROUND AND PURPOSE: Hereditary transthyretin (hATTR) amyloidosis causes progressive polyneuropathy resulting from transthyretin (TTR) amyloid deposition throughout the body, including the peripheral nerves. The efficacy and safety of inotersen, an antisense oligonucleotide inhibitor of TTR prote...

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Vydáno v:Eur J Neurol
Hlavní autoři: Brannagan, T. H., Wang, A. K., Coelho, T., Waddington Cruz, M., Polydefkis, M. J., Dyck, P. J., Plante‐Bordeneuve, V., Berk, J. L., Barroso, F., Merlini, G., Conceição, I., Hughes, S. G., Kwoh, J., Jung, S. W., Guthrie, S., Pollock, M., Benson, M. D., Gertz, M.
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2020
Témata:
Neuropathies
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7496583/
https://ncbi.nlm.nih.gov/pubmed/32343462
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/ene.14285
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