E-LIB Suche - Ergebnisse für: Boespflug-Tanguy, Odile

1

Neuroglobin overexpression in cerebellar neurons of Harlequ..:

Cwerman-Thibault, Hélène ; Malko-Baverel, Vassilissa ; Le Guilloux, Gwendoline...
Molecular Therapy. , 2024

Link: https://doi.org/10.1016/..

2

Safety and efficacy of givinostat in boys with Duchenne mus..:

Mercuri, Eugenio ; Vilchez, Juan J ; Boespflug-Tanguy, Odile...
The Lancet Neurology. 23 (2024) 4 - p. 393-403 , 2024

Link: https://doi.org/10.1016/..

3

Questioning cognitive heterogeneity and intellectual functi..:

Kerdreux, Eliot ; Fraize, Justine ; Garzón, Pauline...
The Clinical Neuropsychologist. , 2023

Link: https://doi.org/10.1080/..

4

SUNFISH Parts 1 and 2: 4-year Efficacy and Safety Data of R..:

Servais, Laurent ; Oskoui, Maryam ; Day, John...
Neurology. 100 (2023) 17_supplement_2 - p. , 2023

Link: https://doi.org/10.1212/..

5

Childhood cerebral adrenoleukodystrophy (CCALD) in France: ..:

Sevin, Caroline ; Hatteb, Samira ; Clément, Aurore...
Orphanet Journal of Rare Diseases. 18 (2023) 1 - p. , 2023

Link: https://doi.org/10.1186/..

6

FIREFISH Parts 1 and 2: 36-month safety and efficacy of ris..:

Darras, Basil ; Baranello, Giovanni ; Boespflug-Tanguy, Odile...
Neurology. 100 (2023) 17_supplement_2 - p. , 2023

Link: https://doi.org/10.1212/..

7

Prospective Multicenter Validation of a Simple Blood Test f..:

Mochel, Fanny ; Gras, Domitille ; Luton, Marie-Pierre...
Neurology. 100 (2023) 23 - p. , 2023

Link: https://doi.org/10.1212/..

8

RINT1 deficiency disrupts lipid metabolism and underlies a ..:

Launay, Nathalie ; Ruiz, Montserrat ; Planas-Serra, Laura...
Journal of Clinical Investigation. 133 (2023) 14 - p. , 2023

Link: https://doi.org/10.1172/..

9

Sunfish parts 1 and 2: 4-year efficacy and safety data of r..:

Oskoui, Maryam ; Deconinck, Nicolas ; Mazzone, Elena S....
Journal of the Neurological Sciences. 455 (2023) - p. 121096 , 2023

Link: https://doi.org/10.1016/..

10

Child-to-adult transition: a survey of current practices wi..:

Nanetti, Lorenzo ; Kearney, Mary ; Boesch, Sylvia...
Neurological Sciences. , 2023

Link: https://doi.org/10.1007/..

11

Gene therapy for neurodegenerative disorders in children: d..:

Boespflug-Tanguy, Odile ; Sevin, Caroline ; Piguet, Francoise
Archives de Pédiatrie. 30 (2023) 8 - p. 8S32-8S40 , 2023

Link: https://doi.org/10.1016/..

12

Parents' dilemma: A therapeutic decision for children with ..:

Boursange, Sophie ; Araneda, Marco ; Stalens, Caroline...
Frontiers in Pediatrics. 10 (2022) - p. , 2022

Link: https://doi.org/10.3389/..

13

Movement disorders in MCT8 deficiency/Allan-Herndon-Dudley ..:

Masnada, Silvia ; Sarret, Catherine ; Antonello, Clara Eleonora...
Molecular Genetics and Metabolism. 135 (2022) 1 - p. 109-113 , 2022

Link: https://doi.org/10.1016/..

14

SUNFISH: 3-year Efficacy and Safety of Risdiplam in Types 2..:

Day, John W. ; Deconinck, Nicolas ; Kirschner, Janbernd...
Neurology. 98 (2022) 18_supplement - p. , 2022

Link: https://doi.org/10.1212/..

15

Case Report: A New Family With Pontocerebellar Hypoplasia 1..:

Amin, Mutaz ; Vignal, Cedric ; Hamed, Ahlam A. A....
Frontiers in Genetics. 13 (2022) - p. , 2022

Link: https://doi.org/10.3389/..