Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomise...
Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study
About this item
Full title
Author / Creator
Klopstock, Thomas , Tricta, Fernando , Neumayr, Lynne , Karin, Ivan , Zorzi, Giovanna , Fradette, Caroline , Kmieć, Tomasz , Büchner, Boriana , Steele, Hannah E , Horvath, Rita , Chinnery, Patrick F , Basu, Anna , Küpper, Clemens , Neuhofer, Christiane , Kálmán, Bernadette , Dušek, Petr , Yapici, Zuhal , Wilson, Ian , Zhao, Feng , Zibordi, Federica , Nardocci, Nardo , Aguilar, Christine , Hayflick, Susan J , Spino, Michael , Blamire, Andrew M , Hogarth, Penelope and Vichinsky, Elliott
Publisher
England: Elsevier Ltd
Journal title
Language
English
Formats
Publication information
Publisher
England: Elsevier Ltd
Subjects
More information
Scope and Contents
Contents
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.
We did an 18-month, randomised, double-blind, placebo-controlled trial (TIRCON2012V...
Alternative Titles
Full title
Safety and efficacy of deferiprone for pantothenate kinase-associated neurodegeneration: a randomised, double-blind, controlled trial and an open-label extension study
Authors, Artists and Contributors
Author / Creator
Tricta, Fernando
Neumayr, Lynne
Karin, Ivan
Zorzi, Giovanna
Fradette, Caroline
Kmieć, Tomasz
Büchner, Boriana
Steele, Hannah E
Horvath, Rita
Chinnery, Patrick F
Basu, Anna
Küpper, Clemens
Neuhofer, Christiane
Kálmán, Bernadette
Dušek, Petr
Yapici, Zuhal
Wilson, Ian
Zhao, Feng
Zibordi, Federica
Nardocci, Nardo
Aguilar, Christine
Hayflick, Susan J
Spino, Michael
Blamire, Andrew M
Hogarth, Penelope
Vichinsky, Elliott
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_journals_2239146956
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2239146956
Other Identifiers
ISSN
1474-4422
E-ISSN
1474-4465
DOI
10.1016/S1474-4422(19)30142-5
