TY - JOUR
AU1 - Mundayat, Rajiv
AU2 - Stewart, Michelle
AU3 - Alvir, Jose
AU4 - Short, Sarah
AU5 - Ong, Moh-Lim
AU6 - Keohane, Denis
AU7 - Rill, Denise
AU8 - Sultan, Marla
AB - 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 
TI - 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)
JF - Neurology and Therapy
DO - 10.1007/s40120-018-0097-9
DA - 2018-04-09
UR - https://www.deepdyve.com/lp/springer-journals/positive-effectiveness-of-tafamidis-in-delaying-disease-progression-in-k96f6KWL40
SP - 87
EP - 101
VL - 7
IS - 1
DP - DeepDyve
ER -