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

Klopstock, Thomas; Tricta, Fernando; Neumayr, Lynne; Karin, Ivan; Zorzi, Giovanna; Fradette, Caroline; Kmiec, Tomasz; Buchner, Boriana; Steele, Hannah; Horvath, Rita; Chinnery, Patrick; Basu, Anna; Kupper, Clemens; Neuhofer, Christiane; Kalman, Bernadette; Dusek, Petr; Yapici, ZÜHAL; Wilson, Ian; Zhao, Feng; Zibordi, Federica; Nardocci, Nardo; Aguilar, Christine; Hayflick, Susan; Spino, Michael; Blamire, Andrew; Hogarth, Penelope; Vichinsky, Elliott

doi:10.1016/s1474-4422(19)30142-5

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, Scopus)

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.