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

Klopstock T., Tricta F., Neumayr L., Karin I., Zorzi G., Fradette C., ...More

LANCET NEUROLOGY, vol.18, no.7, pp.631-642, 2019 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 18 Issue: 7
  • Publication Date: 2019
  • Doi Number: 10.1016/s1474-4422(19)30142-5
  • Journal Name: LANCET NEUROLOGY
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Page Numbers: pp.631-642
  • Istanbul University Affiliated: Yes

Abstract

Background 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.