Marcelo Rugiero

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

Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy

Teresa Coelho, Wilson Marques, Noel R. Dasgupta et al. · JAMA · 2023 · 207 citations · Full text

Medicine, Neurology, Pharmacotherapy +16

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 · 184 citations · Full text

Veterinary Medicine, Internal Medicine, Pharmacotherapy +3

Immune-mediated conditions and antibodies associated with sporadic inclusion body myositis

Boyd M. Koffman, Marcelo Rugiero, Marinos C. Dalakas · Muscle & Nerve · 1998 · 116 citations

Life Sciences, Immunology, Neurology +17

Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update

Angela Dispenzieri, Teresa Coelho, Isabel Conceição et al. · Orphanet Journal of Rare Diseases · 2022 · 89 citations · Full text

Life Sciences, Genetics, Molecular Genetics +13