Kolexia
Sevin Caroline Pédiatrie
Sevin Caroline
Pédiatrie
Hôpital Bicêtre
Le Kremlin-Bicêtre, France
121 Activités
73 Followers
Scientifique
Digital
Production scientifique
Activités par an
Expertise
Sujets de recherche
{{person.topmesh1.name}} Leucodystrophie métachromatique Adrénoleucodystrophie Maladies lysosomiales Maladies de Niemann-Pick Démence de Pick Mucopolysaccharidose de type III Maladie de Niemann-Pick de type C Effets secondaires indésirables des médicaments Mucopolysaccharidoses

Collaborateurs

Voir plus
Aubourg Patrick
Aubourg Patrick
Hôpital Bicêtre, Le Kremlin-Bicêtre, France
Cartier-Lacave Nathalie
Cartier-Lacave Nathalie
Gpe Hosp Cochin Saint Vincent De Paul, Paris, France
Mochel Fanny
Mochel Fanny
Hôpital Pitie Salpetriere, Paris, France
Heron-Longe Benedicte
Heron-Longe Benedicte
Hôpital Armand Trousseau, Paris, France

Industries

Voir plus
ORCHARD THERAPEUTICS
25 collaboration(s)
Dernière en 2023
Bluebird
8 collaboration(s)
Dernière en 2021
PTC Therapeutics
6 collaboration(s)
Dernière en 2020
Takeda Pharmaceutical
2 collaboration(s)
Dernière en 2022

Dernières activités

Voir plus
Newborn screening in metachromatic leukodystrophy – European consensus-based recommendations on clinical management
European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society   09 mars 2024
Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries.
Orphanet journal of rare diseases   07 février 2024
Cell-based device provides effective therapeutic strategy to treat metachromatic leukodystrophy
Molecular genetics and metabolism   01 février 2024
A phase 2 study assessing TAK-611150 mg intrathecal weekly in patients with late-infantile metachromatic leukodystrophy (SHP611–201; EMBOLDEN) compared to matched historical control data from children with late-infantile MLD (GLIA-MLD)
Molecular genetics and metabolism   01 février 2024
Intravenous gene therapy using AAVPHP.eB for metachromatic leukodystrophy
Molecular genetics and metabolism   01 février 2024
MATRIX - "Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia"
Essai Clinique (Assistance publique – Hôpitaux de Paris)   08 janvier 2024
Gene therapy for neurodegenerative disorders in children: dreams and realities.
Archives de pediatrie : organe officiel de la Societe francaise de pediatrie   05 décembre 2023
Childhood cerebral adrenoleukodystrophy (CCALD) in France: Epidemiology, natural history, and burden–A real-world study using registry data linked to the French national claims database
Abstracts of ICPE 2023, the 39th international conference on pharmacoepidemiology and therapeutic risk management (ICPE), 25–27 August 2023, Halifax, Canada   12 octobre 2023
Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden of disease - A population-based study.
Orphanet journal of rare diseases   10 août 2023
Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study.
Orphanet journal of rare diseases   21 juillet 2023