Federica Zibordi

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

Thomas Klopstock, Fernando Tricta, Lynne Neumayr et al. · The Lancet Neurology · 2019 · 146 citations

Medicine, Pharmacology, Pharmacotherapy +11

Iron‐related MRI images in patients with pantothenate kinase–associated neurodegeneration (PKAN) treated with deferiprone: Results of a phase II pilot trial

Giovanna Zorzi, Federica Zibordi, Luisa Chiapparini et al. · Movement Disorders · 2011 · 140 citations

Medicine, Neurology, Pharmacotherapy +17

Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial

Stefan Groeneweg, Robin P. Peeters, Carla Moran et al. · The Lancet Diabetes & Endocrinology · 2019 · 126 citations · Full text

Medicine, Endocrinology, Pharmacology +4

Metabolic consequences of mitochondrial coenzyme A deficiency in patients with PANK2 mutations

Valerio Leoni, Laura Strittmatter, Giovanna Zorzi et al. · Molecular Genetics and Metabolism · 2011 · 120 citations · Full text

Life Sciences, Biochemistry, Genetics +11

Disease characteristics of MCT8 deficiency: an international, retrospective, multicentre cohort study

Stefan Groeneweg, Ferdy S. van Geest, Ayhan Abacı et al. · The Lancet Diabetes & Endocrinology · 2020 · 113 citations · Full text

Life Sciences, Endocrinology, Immunology +3