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Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study

dc.contributor.authorHorvath, Rita
dc.contributor.authorKlopstock, Thomas
dc.contributor.authorTricta, Fernando
dc.contributor.authorNeumayr, Lynne
dc.contributor.authorKarin, Ivan
dc.contributor.authorZorzi, Giovanna
dc.contributor.authorFradette, Caroline
dc.contributor.authorKmiec, Tomasz
dc.contributor.authorBuchner, Boriana
dc.contributor.authorSteele, Hannah E.
dc.contributor.authorChinnery, Patrick F.
dc.contributor.authorBasu, Anna
dc.contributor.authorKupper, Clemens
dc.contributor.authorNeuhofer, Christiane
dc.contributor.authorKalman, Bernadette
dc.contributor.authorDusek, Petr
dc.contributor.authorWilson, Ian
dc.contributor.authorZhao, Feng
dc.contributor.authorZibordi, Federica
dc.contributor.authorNardocci, Nardo
dc.contributor.authorAguilar, Christine
dc.contributor.authorHayflick, Susan J.
dc.contributor.authorSpino, Michael
dc.contributor.authorBlamire, Andrew M.
dc.contributor.authorHogarth, Penelope
dc.contributor.authorVichinsky, Elliott
dc.contributor.authorYapici, Zuhal
dc.date.accessioned2021-03-04T18:51:33Z
dc.date.available2021-03-04T18:51:33Z
dc.date.issued2019
dc.identifier.citationKlopstock T., Tricta F., Neumayr L., Karin I., Zorzi G., Fradette C., Kmiec T., Buchner B., Steele H. E. , Horvath R., et al., "Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study", LANCET NEUROLOGY, cilt.18, ss.631-642, 2019
dc.identifier.issn1474-4422
dc.identifier.othervv_1032021
dc.identifier.otherav_8ce53507-8e35-489c-a4a3-b18db49fb0d0
dc.identifier.urihttp://hdl.handle.net/20.500.12627/95289
dc.identifier.urihttps://doi.org/10.1016/s1474-4422(19)30142-5
dc.description.abstractBackground Pantothenate kinase-associated neurodegeneration (PKAN) is a rare genetic disorder characterised by progressive generalised dystonia and brain iron accumulation. We assessed whether the iron chelator deferiprone can reduce brain iron and slow disease progression.
dc.language.isoeng
dc.subjectDahili Tıp Bilimleri
dc.subjectKLİNİK NEUROLOJİ
dc.subjectKlinik Tıp
dc.subjectKlinik Tıp (MED)
dc.subjectTıp
dc.subjectSağlık Bilimleri
dc.subjectNöroloji
dc.titleSafety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study
dc.typeMakale
dc.relation.journalLANCET NEUROLOGY
dc.contributor.departmentUniversity of Munich , ,
dc.identifier.volume18
dc.identifier.issue7
dc.identifier.startpage631
dc.identifier.endpage642
dc.contributor.firstauthorID266018


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