Patisiran: First Global Approval
Sign Up to like & getrecommendations! Published in 2018 at "Drugs"
DOI: 10.1007/s40265-018-0983-6
Abstract: Patisiran (ONPATTRO™) is a double-stranded small interfering RNA encapsulated in a lipid nanoparticle for delivery to hepatocytes. By specifically binding to a genetically conserved sequence in the 3′ untranslated region of mutant and wild-type transthyretin… read more here.
Keywords: patisiran; ttr; global approval; patisiran first ... See more keywords
Impact of Patisiran, an RNAi Therapeutic, on Orthostatic Intolerance in Patients with Hereditary Transthyretin-Mediated Amyloidosis
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DOI: 10.1016/j.cardfail.2019.07.222
Abstract: Hereditary transthyretin-mediated (hATTR) amyloidosis is a rapidly progressive, multisystem disease which can impact numerous organs, including peripheral and autonomic nerves and heart. Orthostatic intolerance (OI), caused by amyloid deposition in the autonomic nervous system, is… read more here.
Keywords: impact; hereditary transthyretin; transthyretin mediated; hattr amyloidosis ... See more keywords
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
A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
Sign Up to like & getrecommendations! Published in 2020 at "Orphanet Journal of Rare Diseases"
DOI: 10.1186/s13023-020-01399-4
Abstract: Background Patisiran, an RNA interference therapeutic, has demonstrated robust reduction of wild-type and mutant transthyretin protein and was able to improve polyneuropathy and quality of life following 18 months of treatment in patients with hereditary transthyretin-mediated… read more here.
Keywords: patisiran; treatment; treatment patients; study ... See more keywords