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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

dc.contributor.authorCoelho, Teresa
dc.contributor.authorAdams, David
dc.contributor.authorPolydefkis, Michael
dc.contributor.authorGonzalez-Duarte, Alejandra
dc.contributor.authorWixner, Jonas
dc.contributor.authorKristen, Arnt
dc.contributor.authorWhite, Matthew T.
dc.contributor.authorVest, John
dc.contributor.authorBerber, Erhan
dc.contributor.authorSweetser, Marianne T.
dc.contributor.authorSchmidt, Hartmut H.
dc.contributor.authorBerk, John L.
dc.contributor.authorLosada Lopez, Ines Asuncion
dc.contributor.authorDispenzieri, Angela
dc.contributor.authorQuan, Dianna
dc.contributor.authorConceicao, Isabel M.
dc.contributor.authorSlama, Michel S.
dc.contributor.authorGillmore, Julian D.
dc.contributor.authorKyriakides, Theodoros
dc.contributor.authorAjroud-Driss, Senda
dc.contributor.authorWaddington-Cruz, Marcia
dc.contributor.authorMezei, Michelle M.
dc.contributor.authorPlante-Bordeneuve, Violaine
dc.contributor.authorAttarian, Shahram
dc.contributor.authorMauricio, Elizabeth
dc.contributor.authorBrannagan, Thomas H.
dc.contributor.authorUeda, Mitsuharu
dc.contributor.authorAldinc, Emre
dc.contributor.authorWang, Jing Jing
dc.date.accessioned2022-02-18T10:22:48Z
dc.date.available2022-02-18T10:22:48Z
dc.date.issued2021
dc.identifier.citationAdams D., Polydefkis M., Gonzalez-Duarte A., Wixner J., Kristen A., Schmidt H. H. , Berk J. L. , Losada Lopez I. A. , Dispenzieri A., Quan D., et al., "Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study", LANCET NEUROLOGY, cilt.20, sa.1, ss.49-59, 2021
dc.identifier.issn1474-4422
dc.identifier.otherav_96815bf1-9794-41a5-aa7b-26bb4b8b16bf
dc.identifier.othervv_1032021
dc.identifier.urihttp://hdl.handle.net/20.500.12627/179130
dc.identifier.urihttps://doi.org/10.1016/s1474-4422(20)30368-9
dc.description.abstractBackground 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 TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy.
dc.language.isoeng
dc.subjectNöroloji
dc.subjectHealth Sciences
dc.subjectLife Sciences
dc.subjectNeurology (clinical)
dc.subjectDahili Tıp Bilimleri
dc.subjectNeurology
dc.subjectKLİNİK NEUROLOJİ
dc.subjectKlinik Tıp
dc.subjectKlinik Tıp (MED)
dc.subjectTıp
dc.subjectSağlık Bilimleri
dc.titleLong-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
dc.typeMakale
dc.relation.journalLANCET NEUROLOGY
dc.contributor.departmentUniversite Paris Saclay , ,
dc.identifier.volume20
dc.identifier.issue1
dc.identifier.startpage49
dc.identifier.endpage59
dc.contributor.firstauthorID3388187


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