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

1

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/..

2

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/..

3

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/..

4

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/..

5

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/..

6

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/..

7

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/..

8

Questioning cognitive heterogeneity and intellectual functi..:

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

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

9

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/..

10

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/..

11

A novel homozygous mutation in TRAPPC9 gene causing autosom..:

Amin, Mutaz ; Vignal, Cedric ; Eltaraifee, Esraa...
BMC Medical Genomics. 15 (2022) 1 - p. , 2022

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

12

Mutation of Proteolipid Protein 1 Gene: From Severe Hypomye..:

Khalaf, Guy ; Mattern, Claudia ; Begou, Mélina...
Biomedicines. 10 (2022) 7 - p. 1709 , 2022

Link: https://doi.org/10.3390/..

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

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/..

15

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/..