Hani Kushlaf

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

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Benedikt Schoser, Mark Roberts, Barry J. Byrne et al. · The Lancet Neurology · 2021 · 124 citations

Clinical Trials, Cipaglucosidase Alfa, Pharmacotherapy +2

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 · 49 citations · Full text

Genotype–phenotype correlations in valosin-containing protein disease: a retrospective muticentre study

Marianela Schiava, Chiseko Ikenaga, Rocío N. Villar‐Quiles et al. · Journal of Neurology Neurosurgery & Psychiatry · 2022 · 37 citations · Full text

Neurologic and neuroscience education

Stefano Sandrone, Jimmy V. Berthaud, Miguel Chuquilin et al. · Neurology · 2018 · 31 citations

Neuropsychology, Neuropsychiatric Disorders, Neurological Disorder +26