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., ...Daha Fazla

LANCET NEUROLOGY, cilt.18, sa.7, ss.631-642, 2019 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 18 Sayı: 7
  • Basım Tarihi: 2019
  • Doi Numarası: 10.1016/s1474-4422(19)30142-5
  • Dergi Adı: LANCET NEUROLOGY
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus
  • Sayfa Sayıları: ss.631-642
  • İstanbul Üniversitesi Adresli: Evet

Özet

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.