Author Correction: Time-to-event analysis mitigates the impact of symptomatic therapy on therapeutic benefit in Parkinson’s disease trials
Gennaro Pagano, Dylan Trundell, Tanya Simuni, Nicola Pavese et autres
0 citationsnpj Parkinson s Disease
Cecile Paquet Luzy
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22Publications signalées
777Citations signalées
2Affiliations récentes
Institutions déclarées
Domaines associés
Lysosomal Storage Disorders ResearchGlycosylation and Glycoproteins ResearchChild Nutrition and Feeding IssuesGalectins and Cancer BiologyCarbohydrate Chemistry and Synthesis
Publications récentes
Exploratory digital outcome measures of motor sign progression in Parkinson’s disease patients treated with prasinezumab
Kirsten I. Taylor, Florian Lipsmeier, Marzia A. Scelsi, Ekaterina Volkova-Volkmar et autres
14 citationsnpj Digital Medicine
A phase 2 clinical study in Niemann-Pick disease type C and GM2 gangliosidosis patients (RAINBOW)
Roberto Giugliani, Daniel A.D. Valle, Dafne Dain Gandelman Horovitz, Ana María Martins et autres
0 citationsMolecular Genetics and Metabolism
A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Patients' and caregivers' assessments
Roberto Giugliani, Paul Harmatz, Bénédicte Héron, Marc C. Patterson et autres
0 citationsMolecular Genetics and Metabolism
A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Baseline clinical data
Roberto Giugliani, Paul Harmatz, Bénédicte Héron, Marc C. Patterson et autres
0 citationsMolecular Genetics and Metabolism
Use of wearable sensor technology to identify digital biomarkers for monitoring gait parameters in children and adolescents with GM1 and GM2 gangliosidoses
Renata Medinaceli Quintela, Noemie Hurst, Laura López de Frutos, Aurelien Thiers et autres
0 citationsMolecular Genetics and Metabolism
A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidosis (PRONTO): Evaluation of different assessments
Roberto Giugliani, Paul Harmatz, Bénédicte Héron, Marc C. Patterson et autres
0 citationsMolecular Genetics and Metabolism
First-in-human single-dose study of nizubaglustat, a dual inhibitor of ceramide glucosyltransferase and non-lysosomal glucosylceramidase: Safety, tolerability, pharmacokinetics, and pharmacodynamics of single ascending and multiple doses in healthy adults
Cecile Paquet Luzy, Emilie Doppler, Thomas M. Polasek, R. Giorgino
7 citationsMolecular Genetics and Metabolism
Prospective longitudinal study of neurological disease trajectory in children living with late-infantile or juvenile onset of GM1 or GM2 gangliosidosis (PRONTO study)
Roberto Giugliani, Bénédicte Héron, Jeanine R. Jarnes, Marc C. Patterson et autres
0 citationsMolecular Genetics and Metabolism
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, Gérard Greig et autres
83 citationsPLoS ONE
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, Gérard Greig et autres
5 citationsbioRxiv (Cold Spring Harbor Laboratory)
The cognitive profile of type 1 Gaucher disease patients
Marieke Biegstraaten, Keith Wesnes, Cecile Paquet Luzy, Мilan Petakov et autres
22 citationsJournal of Inherited Metabolic Disease
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