Kolexia
Aubourg Patrick Pédiatrie
Aubourg Patrick
Pédiatrie
Hôpital Bicêtre
Le Kremlin-Bicêtre, France
105 Activités
489 Followers
Scientifique
Digital
Production scientifique
Activités par an
Expertise
Sujets de recherche
{{person.topmesh1.name}} Adrénoleucodystrophie Leucodystrophie métachromatique Maladie d'Alzheimer Maladies neurodégénératives Maladies du système nerveux central Leucoencéphalopathies Ataxie Maladies des dents Dégénérescence nerveuse

Collaborateurs

Voir plus
Cartier-Lacave Nathalie
Cartier-Lacave Nathalie
Gpe Hosp Cochin Saint Vincent De Paul, Paris, France
Sevin Caroline
Sevin Caroline
Hôpital Bicêtre, Le Kremlin-Bicêtre, France
Bieche Ivan
Bieche Ivan
Institut Curie, Saint-Cloud, France
Cavazzana Marina
Cavazzana Marina
Hôpital Necker Enfants Malades, Paris, France

Industries

Voir plus
INC RESEARCH, LLC
1 collaboration(s)
Dernière en 2017
{{person.topindus2.name}}
{{person.topindus2.tot}} collaboration(s)
Dernière en {{person.topindus2.last}}
{{person.topindus3.name}}
{{person.topindus3.tot}} collaboration(s)
Dernière en {{person.topindus3.last}}
{{person.topindus4.name}}
{{person.topindus4.tot}} collaboration(s)
Dernière en {{person.topindus4.last}}

Dernières activités

Voir plus
Biomarker-based risk prediction for the onset of neuroinflammation in X-linked adrenoleukodystrophy.
EBioMedicine   07 septembre 2023
A Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD): A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy (CALD)
Essai Clinique (Bluebird)   25 mars 2022
TG-MLD: A Phase I/II, Open Labeled, Monocentric Study of Direct Intracranial Administration of a Replication Deficient Adeno-associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human ARSA cDNA to Children With Metachromatic Leukodystrophy.
Essai Clinique (Institut National de la Santé Et de la Recherche Médicale, France)   10 janvier 2022
Elivaldogene autotemcel (ELI-CEL; LENTI-D) gene therapy for cerebral adrenoleukodystrophy: updated results from the phase 2/3 study and safety outcomes report from the phase 3 study
Abstracts from the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT)   01 juin 2021
AAV2/9-mediated silencing of PMP22 prevents the development of pathological features in a rat model of Charcot-Marie-Tooth disease 1 A.
Nature communications   21 avril 2021
Neurofilament light chain as a potential biomarker for monitoring neurodegeneration in X-linked adrenoleukodystrophy.
Nature communications   22 mars 2021
Interim results from phase 2/3 (ALD-102) and phase 3 (ALD-104) studies of elivaldogene autotemcel (Lenti-D) gene therapy for the treatment of cerebral adrenoleukodystrophy
British Paediatric Neurology Association, Abstracts of the Annual Meeting, 6–8 January 2021, Virtual   30 décembre 2020
Lenti-D Hematopoietic Stem Cell Gene Therapy Stabilizes Neurologic Function in Boys with Cerebral Adrenoleukodystrophy
Abstracts from the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT)   01 décembre 2020
Safety of Autologous Hematopoietic Stem Cell Transplantation with Gene Addition Therapy for Transfusion-Dependent β-Thalassemia, Sickle Cell Disease, and Cerebral Adrenoleukodystrophy
Abstracts from the 46th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT)   01 décembre 2020
Intra-CSF AAV9 and AAVrh10 Administration in Nonhuman Primates: Promising Routes and Vectors for Which Neurological Diseases?
Molecular therapy. Methods & clinical development   11 avril 2020