Cecile Paquet Luzy

Miglustat in adult and juvenile patients with Niemann–Pick disease type C: Long-term data from a clinical trial

J. E. Wraith, Darleen Vecchio, Elizabeth Jacklin et al. · Molecular Genetics and Metabolism · 2009 · 207 citations

Pediatrics, Clinical Disease, Neuropathology +3

Long-Term Miglustat Therapy in Children With Niemann-Pick Disease Type C

Marc C. Patterson, Darleen Vecchio, Elizabeth Jacklin et al. · Journal of Child Neurology · 2009 · 167 citations

Natural history of Niemann-Pick disease type C in a multicentre observational retrospective cohort study

J. E. Wraith, Nathalie Guffon, Marianne Rohrbach et al. · Molecular Genetics and Metabolism · 2009 · 107 citations

Disease Progression, Inherited Metabolic Disease, Clinical Epidemiology +6

Miglustat in late-onset Tay-Sachs disease: a 12-month, randomized, controlled clinical study with 24 months of extended treatment

Barbara E. Shapiro, Gregory M. Pastores, John G. Gianutsos et al. · Genetics in Medicine · 2009 · 105 citations · Full text

Late-onset Tay-sachs Disease, Pathology, Neurology +2

Test-retest reliability of task-based and resting-state blood oxygen level dependence and cerebral blood flow measures

Štefan Holiga, Fabio Sambataro, Cecile Paquet Luzy et al. · PLoS ONE · 2018 · 77 citations · Full text

Brain Mapping, Brain Circulation, Social Sciences +20