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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

Lookup NU author(s): Dr Hannah Steele, Professor Rita HorvathORCiD, Professor Patrick Chinnery, Dr Anna BasuORCiD, Professor Andrew BlamireORCiD



This work is licensed under a Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License (CC BY-NC-ND).

Publication metadata

Author(s): Klopstock T, Tricta F, Neumayr L, Karin I, Zorzi G, Fradette C, Kmiec T, Buchner B, Steele HR, Horvath R, Chinnery PF, Basu A, Kupper C, Neuhofer C, Kalman B, Dusek P, Yapici Z, Wilson JI, Zhao F, Zibordi F, Nardocci N, Aguilar C, Hayflick SJ, Spino M, Blamire AM, Hogarth P, Vichinsky E

Publication type: Article

Publication status: Published

Journal: Lancet Neurology

Year: 2019

Volume: 18

Pages: 631-642

Print publication date: 01/07/2019

Online publication date: 14/06/2019

Acceptance date: 27/03/2019

Date deposited: 14/06/2019

ISSN (print): 1474-4422

ISSN (electronic): 1474-4422

Publisher: Elsevier Ltd.


DOI: 10.1016/S1474-4422(19)30142-5


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