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

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 of the Pha...

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Vydáno v:	BMC neurology Ročník 17; číslo 1; s. 181 - 12
Hlavní autoři:	Adams, David, Suhr, Ole B., Dyck, Peter J., Litchy, William J., Leahy, Raina G., Chen, Jihong, Gollob, Jared, Coelho, Teresa
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	London BioMed Central 11.09.2017 BioMed Central Ltd Springer Nature B.V BMC
Témata:	Adolescent Adult Aged Aged, 80 and over Amyloidosis Amyloidosis - drug therapy APOLLO Autonomic nervous system Body mass Body Mass Index Clinical trials Diabetes Diabetes mellitus Diabetic Neuropathies - drug therapy Diabetic neuropathy Double-Blind Method Drug therapy hATTR amyloidosis Heart diseases Humans Innervation Intravenous administration Medicine Medicine & Public Health Methods Middle Aged mNIS+7 Morbidity Mutation Nerve conduction Neural Conduction Neurochemistry Neurology Neuromuscular disorders and peripheral neurology Neurosurgery Nutritional status Patients Patisiran Polyneuropathies - drug therapy Polyneuropathy Proteins Quality of Life Reflexes RNA interference RNA, Small Interfering - therapeutic use RNA-mediated interference Study Protocol Surveys and Questionnaires Young Adult β-Amyloid RNA interference hATTR amyloidosis APOLLO Patisiran mNIS+7 Polyneuropathy Methods
ISSN:	1471-2377, 1471-2377
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