Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
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DOI: 10.1016/s1474-4422(20)30368-9
Abstract: BACKGROUND Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits… read more here.
Keywords: apollo patisiran; group; patisiran; phase ole ... See more keywords