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

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

Abstract 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 prog...

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Bibliographic Details
Main Authors: Leslie Amass, Huihua Li, Balarama K. Gundapaneni, Jeffrey H. Schwartz, Denis J. Keohane
Format: Article
Language:English
Published: BMC 2018-12-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Transthyretin
Amyloidosis
Baseline severity
Disease progression
Polyneuropathy
NIS-LL
Online Access:http://link.springer.com/article/10.1186/s13023-018-0947-7

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http://link.springer.com/article/10.1186/s13023-018-0947-7

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