E-LIB Suche - Ergebnisse für: Muntoni, Francesco

1

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/..

2

The Clinical Development of Taldefgrobep Alfa: An Anti-Myos..:

Muntoni, Francesco ; Byrne, Barry J. ; McMillan, Hugh J....
Neurology and Therapy. 13 (2024) 1 - p. 183-219 , 2024

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

3

Quantifying Variability in Motor Function in Duchenne Muscu..:

Stimpson, Georgia ; Ridout, Deborah ; Wolfe, Amy...
Journal of Neuromuscular Diseases. 11 (2024) 1 - p. 153-166 , 2024

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

4

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/..

5

Lower limb muscle MRI fat fraction is a responsive outcome ..:

Doherty, Carolynne M. ; Morrow, Jasper M. ; Zuccarino, Riccardo...
Annals of Clinical and Translational Neurology. 11 (2024) 3 - p. 607-617 , 2024

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

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

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/..

8

Understanding anxiety experienced by young males with Duche..:

Trimmer, Rachel E. ; Mandy, William P.L. ; Muntoni, Francesco.
Neuromuscular Disorders. 34 (2024) - p. 95-104 , 2024

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

9

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/..

10

Changes in abilities over the initial 12 months of nusiners..:

Coratti, Giorgia ; Civitello, Matthew ; Rohwer, Annemarie...
Neuromuscular Disorders. , 2024

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

11

Quantitative Foot Muscle Magnetic Resonance Imaging Reliabl..:

Doherty, Carolynne M. ; Howard, Paige ; O'Donnell, Luke F....
Annals of Neurology. , 2024

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

12

Assessment of the upper limb function, strength, and mobili..:

Milev, Evelin ; Selby, Victoria ; Wolfe, Amy...
Muscle & Nerve. 69 (2024) 3 - p. 340-348 , 2024

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

13

Safety and efficacy of gene replacement therapy for X-linke..:

Shieh, Perry B ; Kuntz, Nancy L ; Dowling, James J...
The Lancet Neurology. 22 (2023) 12 - p. 1125-1139 , 2023

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

14

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/..

15

Networking to Optimize Dmd exon 53 Skipping in the Brain of..:

Doisy, Mathilde ; Vacca, Ophélie ; Fergus, Claire...
Biomedicines. 11 (2023) 12 - p. 3243 , 2023

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