patisiran
National Institutes of Health
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Therapeutic oligonucleotides, such as small interfering RNAs (siRNAs), provide a simple and effective tool to modulate expression…
Impact of Patisiran on overall health status in hATTR amyloidosis : Results from the APOLLO trial
The use of the names for patisiran has been made consistent throughout the article in line with the journal style and…
We thank the authors of the recent letter to the editor [1] for their thoughtful review of our article, ‘An indirect treatment…
Objective: This report describes the efficacy and safety of patisiran from the Phase 3 APOLLO study.
Background: hATTR…
Objective: To assess the impact of patisiran, an investigational RNAi therapeutic, on quality of life and disability among…
Zusammenfassung Die Transthyretin-Amyloidose (ATTR-Amyloidose) ist eine seltene, rasch verlaufende neurodegenerative Erkrankung…
With one full quarter still to go before the end of the year, 41 novel drugs have already been approved by the FDA, putting 2018…
Background:
Familial amyloid polyneuropathy (FAP) is a progressive, fatal disease caused by deposition of transthyretin (TTR…