E-LIB Suche - Ergebnisse für: Mercuri, Eugenio

1

Can the CHOP-INTEND be used as An Outcome Measure in the Fi..:

Cutrona, Costanza ; de Sanctis, Roberto ; Coratti, Giorgia...
Journal of Neuromuscular Diseases. 11 (2024) 1 - p. 85-90 , 2024

Link: https://doi.org/10.3233/..

2

Early neurological signs in infants identified through neon..:

Pane, Marika ; Stanca, Giulia ; Ticci, Chiara...
European Journal of Pediatrics. , 2024

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

3

Practical Considerations for Delandistrogene Moxeparvovec G..:

Mendell, Jerry R. ; Proud, Crystal ; Zaidman, Craig M....
Pediatric Neurology. 153 (2024) - p. 11-18 , 2024

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

4

Neurological assessment tool for screening infants during t..:

Romeo, Domenico M. ; Velli, Chiara ; Sini, Francesca...
Developmental Medicine & Child Neurology. , 2024

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

5

Real-World Outcomes in Patients with Spinal Muscular Atroph..:

Servais, Laurent ; Day, John W. ; De Vivo, Darryl C....
Journal of Neuromuscular Diseases. 11 (2024) 2 - p. 425-442 , 2024

Link: https://doi.org/10.3233/..

6

Bulbar function in spinal muscular atrophy (SMA): State of ..:

McGrattan, Katlyn ; Cerchiari, Antonella ; Conway, Eleanor...
Neuromuscular Disorders. 38 (2024) - p. 44-50 , 2024

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

7

Ligamentous laxity in children with achondroplasia: Prevale..:

Romeo, Domenico Marco ; Pironi, Virginia ; Velli, Chiara...
European Journal of Medical Genetics. 68 (2024) - p. 104930 , 2024

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

8

Survival among patients receiving eteplirsen for up to 8 ye..:

Iff, Joel ; Done, Nicolae ; Tuttle, Edward...
Muscle & Nerve. , 2024

Link: https://doi.org/10.1002/..

9

JEWELFISH: 24-month results from an open-label study in non..:

Chiriboga, Claudia A. ; Bruno, Claudio ; Duong, Tina...
Journal of Neurology. , 2024

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

10

Hospital admissions from the emergency department of adult ..:

Monforte, Mauro ; Torchia, Eleonora ; Bortolani, Sara...
European Journal of Neurology. 31 (2024) 5 - p. , 2024

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

11

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/..

12

JEWELFISH: 24-month Safety, Pharmacodynamic and Exploratory..:

Chiriboga, Claudia ; Bruno, Claudio ; Duong, Tina...
Neurology. 100 (2023) 17_supplement_2 - p. , 2023

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

13

Letter to the Editor: In response to P.R. Clemens et al., E..:

Muntoni, Francesco ; Straub, Volker ; Servais, Laurent.
Journal of Neuromuscular Diseases. 10 (2023) 6 - p. 1151-1153 , 2023

Link: https://doi.org/10.3233/..

14

P260: Survival in eteplirsen-treated vs Duchenne muscular d..:

O'Rourke, Erin ; Iff, Joel ; Done, Nicolae...
Genetics in Medicine Open. 1 (2023) 1 - p. 100288 , 2023

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

15

Safety, tolerability and pharmacokinetics of eteplirsen in ..:

Mercuri, E. ; Seferian, A.M. ; Servais, L....
Neuromuscular Disorders. 33 (2023) 6 - p. 476-483 , 2023

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