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Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)

Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial... Neurol Ther (2018) 7:87–101 https://doi.org/10.1007/s40120-018-0097-9 ORIGINAL RESEARCH Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS) . . . . Rajiv Mundayat Michelle Stewart Jose Alvir Sarah Short . . . Moh-Lim Ong Denis Keohane Denise Rill Marla B. Sultan Received: November 29, 2017 / Published online: April 9, 2018 The Author(s) 2018 Limbs (p \ 0.001) and its subscales (p \ 0.023) ABSTRACT compared with untreated subjects. There was significantly less deterioration among tafamidis- Introduction: The effectiveness of tafamidis for treated subjects compared with untreated sub- the treatment of transthyretin familial amyloid jects on the Norfolk Quality of Life scale polyneuropathy (TTR-FAP) was evaluated using (p \ 0.001). There were no significant differences data from the Transthyretin Amyloidosis Out- observed in functional (assessed by Karnofsky comes Survey (THAOS) registry. Performance Status Scale score) or nutritional Methods: Subjects receiving tafamidis (n = 252) (assessed by modified body mass index) status were compared with untreated subjects in a between the treated and untreated groups. The non-randomized, matched cohort analysis. primary model which examined survival from Subjects were matched with up to four baseline http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Neurology and Therapy Springer Journals

Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)

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Publisher
Springer Journals
Copyright
Copyright © 2018 by The Author(s)
Subject
Medicine & Public Health; Internal Medicine; Neurology
ISSN
2193-8253
eISSN
2193-6536
DOI
10.1007/s40120-018-0097-9
Publisher site
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Abstract

Neurol Ther (2018) 7:87–101 https://doi.org/10.1007/s40120-018-0097-9 ORIGINAL RESEARCH Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS) . . . . Rajiv Mundayat Michelle Stewart Jose Alvir Sarah Short . . . Moh-Lim Ong Denis Keohane Denise Rill Marla B. Sultan Received: November 29, 2017 / Published online: April 9, 2018 The Author(s) 2018 Limbs (p \ 0.001) and its subscales (p \ 0.023) ABSTRACT compared with untreated subjects. There was significantly less deterioration among tafamidis- Introduction: The effectiveness of tafamidis for treated subjects compared with untreated sub- the treatment of transthyretin familial amyloid jects on the Norfolk Quality of Life scale polyneuropathy (TTR-FAP) was evaluated using (p \ 0.001). There were no significant differences data from the Transthyretin Amyloidosis Out- observed in functional (assessed by Karnofsky comes Survey (THAOS) registry. Performance Status Scale score) or nutritional Methods: Subjects receiving tafamidis (n = 252) (assessed by modified body mass index) status were compared with untreated subjects in a between the treated and untreated groups. The non-randomized, matched cohort analysis. primary model which examined survival from Subjects were matched with up to four baseline

Journal

Neurology and Therapy – Springer Journals

Published: Apr 9, 2018

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