Patisiran in hATTR Amyloidosis: Six-Month Latency Period before Efficacy
نویسندگان
چکیده
منابع مشابه
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 Phase 3 APOLLO study, a randomized, double-blind, placebo-controlled, global study to evaluate the...
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ژورنال
عنوان ژورنال: Brain Sciences
سال: 2021
ISSN: 2076-3425
DOI: 10.3390/brainsci11040515