Paulo Victor Sgobbi de Souza

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

Myasthenia Gravis and COVID-19: Clinical Characteristics and Outcomes

Antônio Edvan Camelo-Filho, André Macedo Serafim da Silva, Eduardo de Paula Estephan et al. · Frontiers in Neurology · 2020 · 105 citations · Full text

Life Sciences, Immunology, Internal Medicine +17

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks

Priya S. Kishnani, Jordi Díaz‐Manera, António Toscano et al. · JAMA Neurology · 2023 · 55 citations · Full text

Life Sciences, Internal Medicine, Pharmacotherapy +8

Intragenic variants in the <i>SMN1</i> gene determine the clinical phenotype in 5q spinal muscular atrophy

Rodrigo de Holanda Mendonça, Ciro Matsui, Graziela Jorge Polido et al. · Neurology Genetics · 2020 · 46 citations · Full text

Life Sciences, Genetics, Molecular Genetics +17

Clinical and radiological profile of patients with spinal muscular atrophy type 4

Paulo Victor Sgobbi de Souza, Wladimir Bocca Vieira de Rezende Pinto, Aracelly R.F. Ricarte et al. · European Journal of Neurology · 2020 · 46 citations

Medicine, Genetics, Neurology +17