Matteo Garibaldi

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 · 800 citations

Medicine, Pharmacology, Pharmacotherapy +6

Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial

James F. Howard, Vera Bril, Tuan Vu et al. · The Lancet Neurology · 2021 · 668 citations · Full text

Medicine, Endocrinology, Pharmacotherapy +4

Neutral Lipid Storage Diseases: clinical/genetic features and natural history in a large cohort of Italian patients

E. Pennisi, Marcello Arca, Enrico Bertini et al. · Orphanet Journal of Rare Diseases · 2017 · 93 citations · Full text

Life Sciences, Cardiology, Genetics +17

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 · 79 citations · Full text

Medicine, Pharmacology, Pharmacotherapy +9

New genotype-phenotype correlations in a large European cohort of patients with sarcoglycanopathy

Jorge Alonso‐Pérez, Lidia González‐Quereda, Luca Bello et al. · Brain · 2020 · 76 citations · Full text

Life Sciences, Genetics, Molecular Genetics +16