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

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/..

3

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/..

4

Questioning cognitive heterogeneity and intellectual functi..:

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

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

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

C.2 SUNFISH parts 1 and 2: 4-year efficacy and safety data ..:

Mah, JK ; Oskoui, M ; Day, JW...
Canadian Journal of Neurological Sciences / Journal Canadien des Sciences Neurologiques. 50 (2023) s2 - p. S51-S52 , 2023

Link: https://doi.org/10.1017/..

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

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/..

9

Mirror Movements Due To a TUBB3 Variant:

Desjardins, Clément ; Gras, Domitille ; Trouillard, Oriane...
Pediatric Neurology. 148 (2023) - p. 142-144 , 2023

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

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

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/..

12

Developmental coordination disorder subtypes in children: A..:

Gras, Domitille ; Ploix Maes, Emmanuelle ; Doulazmi, Mohamed...
Developmental Medicine & Child Neurology. 65 (2023) 10 - p. 1332-1342 , 2023

Link: https://doi.org/10.1111/..

13

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/..

14

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/..

15

Titin copy number variations associated with dominant inher..:

Perrin, Aurélien ; Métay, Corinne ; Savarese, Marco...
info:eu-repo/semantics/altIdentifier/doi/10.1136/jmg-2023-109473. , 2023

Link: https://hal.science/hal-..