Josep Gámez

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

Merrill D. Benson, Márcia Waddington‐Cruz, John L. Berk et al. · New England Journal of Medicine · 2018 · 1.4K citations · Full text

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

Subcutaneous immunoglobulin for maintenance treatment in chronic inflammatory demyelinating polyneuropathy (PATH): a randomised, double-blind, placebo-controlled, phase 3 trial

Ivo N. van Schaik, Vera Bril, Nan van Geloven et al. · The Lancet Neurology · 2017 · 258 citations · Full text

Subcutaneous Immunoglobulin, Autoimmune Disease, Chronic Inflammatory +12

Masitinib as an add-on therapy to riluzole in patients with amyotrophic lateral sclerosis: a randomized clinical trial

Jesús S. Mora, Angela Genge, Adriano Chiò et al. · Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration · 2019 · 214 citations · Full text

Confirmation of the Type 2 Myotonic Dystrophy (CCTG) Expansion Mutation in Patients with Proximal Myotonic Myopathy/Proximal Myotonic Dystrophy of Different European Origins: A Single Shared Haplotype Indicates an Ancestral Founder Effect

Linda L. Bachinski, Bjarne Udd, G. Meola et al. · The American Journal of Human Genetics · 2003 · 139 citations · Full text

Mendelian Disorder, Genetic Disorder, Medicine +7