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
[de novo hATTR amyloidosis after domino transplantation of a donor's liver: a case report for the use of Patisiran].
Sign Up to like & getrecommendations! Published in 2022 at "Zeitschrift fur Gastroenterologie"
DOI: 10.1055/a-1735-3795
Abstract: Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, rapidly progressing, and potentially life-threatening disease caused by one of more than 120 mutations in the transthyretin (TTR) gene. As a result of the cumulative amyloid deposits, especially… read more here.
Keywords: liver; hattr amyloidosis; transplantation; case ... See more keywords
Impact of Inotersen on Functioning and Activities of Daily Living for Patients with Hereditary TTR Amyloidosis: Results from a Double-Blind Placebo-Controlled Trial
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DOI: 10.1182/blood-2018-99-119578
Abstract: Background: Hereditary transthyretin amyloidosis (hATTR) is a rare, systemic, progressive, and fatal condition in which misfolded proteins, mainly produced in the liver, deposit in muscle and organ tissues leading to symptoms of peripheral neuropathy, and… read more here.
Keywords: impact; substantial impairment; placebo; consultancy ... 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 in hATTR Amyloidosis: Six-Month Latency Period before Efficacy
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DOI: 10.3390/brainsci11040515
Abstract: Hereditary amyloidosis associated with mutations in the transthyretin gene (hATTR) is a progressive devastating disease, with a fatal outcome occurring within 10years after onset. In recent years, TTR gene silencing therapy appeared as a promising… read more here.
Keywords: month latency; amyloidosis; latency period; hattr amyloidosis ... See more keywords