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Boespflug-Tanguy, Odile
389  results:
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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8

Questioning cognitive heterogeneity and intellectual functi..:

Kerdreux, Eliot ; Fraize, Justine ; Garzón, Pauline...
The Clinical Neuropsychologist.  , 2023
Link: https://doi.org/10.1080/..
 
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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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/..
 
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