Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy

BACKGROUND: Emerging evidence suggests that several factors can impact disease progression in transthyretin amyloid polyneuropathy (ATTR-PN). The present analysis used longitudinal data from Val30Met patients participating in the tafamidis (selective TTR stabilizer) clinical development program to e...

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Bibliografske podrobnosti
izdano v:Orphanet J Rare Dis
Main Authors: Amass, Leslie, Li, Huihua, Gundapaneni, Balarama K., Schwartz, Jeffrey H., Keohane, Denis J.
Format: Artigo
Jezik:Inglês
Izdano: BioMed Central 2018
Teme:
Research
Online dostop:https://ncbi.nlm.nih.gov/pmc/articles/PMC6296038/
https://ncbi.nlm.nih.gov/pubmed/30558645
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0947-7
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