Kolexia
Chabrol Brigitte
Pédiatrie
Hôpital La Timone Enfants
Marseille, France
434 Activités
781 Followers

Scientifique
Digital
Production scientifique
Activités par an
Expertise
Sujets de recherche
{{person.topmesh1.name}} Maladies rares Urgences Épilepsie Amyotrophie spinale Atrophie Dystrophies musculaires Myopathie de Duchenne Paralysie cérébrale Maladies mitochondriales

Industries

Biogen
3 collaboration(s)
Dernière en 2021
ORCHARD THERAPEUTICS
2 collaboration(s)
Dernière en 2020
Sarepta
2 collaboration(s)
Dernière en 2021
AveXis
2 collaboration(s)
Dernière en 2021

Dernières activités

PHENO SMART: Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Essai Clinique (Hospices Civils de Lyon)   14 mars 2024
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis: Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis
Essai Clinique (argenx)   13 mars 2024
Continuous Deep Sedation Until Death of Children at the End of Life: French Physicians' Opinions.
Journal of palliative medicine   14 février 2024
COMPASS: A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of DNL310 vs Idursulfase in Pediatric Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II
Essai Clinique (Denali Therapeutics Inc.)   05 février 2024
A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Baseline clinical data
Molecular genetics and metabolism   01 février 2024
Performance measures and patient/caregiver-reported assessments collected in a longitudinal, multi-center disease monitoring program (DMP) of patients with mucopolysaccharidosis type VII (MPS VII)
Molecular genetics and metabolism   01 février 2024
A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidosis (PRONTO): Evaluation of different assessments
Molecular genetics and metabolism   01 février 2024
Vestronidase alfa for the treatment of mucopolysaccharidosis type VII (MPS VII): Updated results from a novel, longitudinal, multi-center disease monitoring program (DMP)
Molecular genetics and metabolism   01 février 2024
A natural history study of late-infantile and juvenile GM1 and GM2 gangliosidoses (PRONTO): Patients' and caregivers' assessments
Molecular genetics and metabolism   01 février 2024
MITOMICS: Interest of Multi-omics (WES / RNA-Seq) Approach to Fight Against the Diagnostic Deadlock in Mitochondrial Diseases
Essai Clinique (CHU Nice)   11 janvier 2024