Sevim Erdem‐Özdamar

Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study

James F. Howard, Kimiaki Utsugisawa, Michael Benatar et al. · The Lancet Neurology · 2017 · 745 citations

Multicentre Study, Pharmacology, Medicine +3

Mutations in a Gene Encoding a Novel SH3/TPR Domain Protein Cause Autosomal Recessive Charcot-Marie-Tooth Type 4C Neuropathy

Jan Senderek, Carsten Bergmann, Claudia Stendel et al. · The American Journal of Human Genetics · 2003 · 210 citations · Full text

Neurodegenerative Diseases, Molecular Neuroscience, Mendelian Disorder +9

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Jordi Díaz‐Manera, Priya S. Kishnani, Hani Kushlaf et al. · The Lancet Neurology · 2021 · 169 citations · Full text

Late-onset Pompe Disease, Autoimmune Disease, Clinical Infectious Disease +3

Late onset muscular dystrophy with cerebral white matter changes due to partial merosin deficiency

Ersin Tan, Haluk Topaloğlu, Caroline A. Sewry et al. · Neuromuscular Disorders · 1997 · 79 citations

Neurological Disorder, Corticobasal Degeneration, White Matter +10

Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension

Renato Mantegazza, Gil I. Wolfe, Srikanth Muppidi et al. · Neurology · 2020 · 74 citations · Full text