E-LIB Suche - Ergebnisse für: Eugenio Mercuri

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

Functional trajectories before and after loss of ambulation..:

McDonald, Craig M. ; Signorovitch, James ; Mercuri, Eugenio...
PLOS ONE. 19 (2024) 6 - p. e0304099 , 2024

Link: https://doi.org/10.1371/..

3

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/..

4

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

Iff, Joel ; Done, Nicolae ; Tuttle, Edward...
Muscle & Nerve. 70 (2024) 1 - p. 60-70 , 2024

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

5

A Systematic Literature Review of the Natural History of Re..:

Martí, Yasmina ; Aponte Ribero, Valerie ; Batson, Sarah...
Journal of Neuromuscular Diseases. , 2024

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

6

Meaningful changes in motor function in Duchenne muscular d..:

Muntoni, Francesco ; Signorovitch, James ; Sajeev, Gautam...
PLOS ONE. 19 (2024) 7 - p. e0304984 , 2024

Link: https://doi.org/10.1371/..

7

2024 update: European consensus statement on gene therapy f..:

Kirschner, Janbernd ; Bernert, Günther ; Butoianu, Nina...
European Journal of Paediatric Neurology. 51 (2024) - p. 73-78 , 2024

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

8

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/..

9

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/..

10

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/..

11

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/..

12

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/..

13

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/..

14

Early neurological signs in infants identified through neon..:

Pane, Marika ; Stanca, Giulia ; Ticci, Chiara...
European Journal of Pediatrics. 183 (2024) 7 - p. 2995-2999 , 2024

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

15

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/..