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Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis

Hereditary transthyretin-mediated amyloidosis is a rapidly progressive, heterogeneous disease caused by the accumulation of misfolded transthyretin protein as amyloid fibrils at multiple sites, and is characterized by peripheral sensorimotor neuropathy, autonomic neuropathy and/or cardiomyopathy. Cu...

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Hauptverfasser: Kristen, Arnt (VerfasserIn) , Ajroud-Driss, Senda (VerfasserIn) , Conceição, Isabel (VerfasserIn) , Gorevic, Peter (VerfasserIn) , Kyriakides, Theodoros (VerfasserIn) , Obici, Laura (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 27 Nov 2018
In: Neurodegenerative disease management
Year: 2018, Jahrgang: 9, Heft: 1, Pages: 5-23
ISSN:1758-2024
DOI:10.2217/nmt-2018-0033
Online-Zugang:Verlag, Volltext: https://doi.org/10.2217/nmt-2018-0033
Verlag: https://www.futuremedicine.com/doi/10.2217/nmt-2018-0033
Volltext
Verfasserangaben:Arnt V. Kristen, Senda Ajroud-Driss, Isabel Conceição, Peter Gorevic, Theodoros Kyriakides, & Laura Obici
Beschreibung
Zusammenfassung:Hereditary transthyretin-mediated amyloidosis is a rapidly progressive, heterogeneous disease caused by the accumulation of misfolded transthyretin protein as amyloid fibrils at multiple sites, and is characterized by peripheral sensorimotor neuropathy, autonomic neuropathy and/or cardiomyopathy. Current treatment options have limited efficacy and often do not prevent disease progression. Patisiran is a novel RNA interference therapeutic that specifically reduces production of both wild-type and mutant transthyretin protein. In Phase II, III and long-term extension studies in patients with hereditary transthyretin-mediated amyloidosis, patisiran has consistently slowed or improved progression of neuropathy. In addition, the Phase III trial demonstrated significant improvements in quality of life measures and indicators of cardiomyopathy. Here, we highlight efficacy and safety data from the patisiran clinical trial programme.
Beschreibung:Gesehen am 30.10.2019
Beschreibung:Online Resource
ISSN:1758-2024
DOI:10.2217/nmt-2018-0033

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