Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy

Trial design and rationale for APOLLO, a Phase 3, placebo-controlled study of patisiran in patients with hereditary ATTR amyloidosis with polyneuropathy

Abstract Background Patisiran is an investigational RNA interference (RNAi) therapeutic in development for the treatment of hereditary ATTR (hATTR) amyloidosis, a progressive disease associated with significant disability, morbidity, and mortality. Methods Here we describe the rationale and design o...

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Bibliographic Details
Main Authors: David Adams, Ole B. Suhr, Peter J. Dyck, William J. Litchy, Raina G. Leahy, Jihong Chen, Jared Gollob, Teresa Coelho
Format: Article
Language:English
Published: BMC 2017-09-01
Series:BMC Neurology
Subjects:
Patisiran
APOLLO
RNA interference
hATTR amyloidosis
mNIS+7
Methods
Online Access:http://link.springer.com/article/10.1186/s12883-017-0948-5

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http://link.springer.com/article/10.1186/s12883-017-0948-5

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