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
Sign Up to like & getrecommendations! Published in 2019 at "Journal of Cardiac Failure"
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
Sign Up to like & getrecommendations! Published in 2021 at "The Lancet Neurology"
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
Patisiran exposure in early pregnancy: a case report
Sign Up to like & getrecommendations! Published in 2024 at "Therapeutic Advances in Neurological Disorders"
DOI: 10.1177/17562864241239755
Abstract: We describe here the first case of exposure to patisiran treatment, a small interfering RNA molecule, during early pregnancy of a 36-year-old woman with symptomatic hereditary transthyretin-related amyloidosis. There were no major complications during pregnancy… read more here.
Keywords: early pregnancy; treatment; vitamin; pregnancy ... 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
Patisiran for the treatment of patients with p.Ile88Leu hereditary transthyretin amyloidosis: an Italian real-life experience
Sign Up to like & getrecommendations! Published in 2024 at "Frontiers in Neurology"
DOI: 10.3389/fneur.2024.1415851
Abstract: Objectives Evidence on the activity of patisiran therapy in specific subgroups of patients with hereditary transthyretin amyloidosis variant (ATTRv) is still scarce. This prospective real-world study was designed to provide the first in-depth clinical data… read more here.
Keywords: patisiran treatment; neurology; transthyretin amyloidosis; hereditary transthyretin ... See more keywords