E-LIB Suche - Ergebnisse für: Byrne, Barry J.

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

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

In memoriam: Kenneth I. Berns, MD, PhD (1938–2024):

Byrne, Barry J. ; Flotte, Terence R. ; Herzog, Roland W..
Molecular Therapy. 32 (2024) 4 - p. 867-868 , 2024

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

4

NEO1/NEO-EXT studies: Long-term muscle quantitative magneti..:

Byrne, Barry ; Carlier, Pierre G. ; Vissing, John...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107783 , 2024

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

5

A plain language summary of AAV-based gene therapy: challen..:

Moroz, Tara ; Schulz, Martin ; Levy, Daniel I....
Future Rare Diseases. 4 (2024) 1 - p. , 2024

Link: https://doi.org/10.2217/..

6

Switching treatment to cipaglucosidase alfa plus miglustat ..:

Toscano, Antonio ; Byrne, Barry J. ; Claeys, Kristl G....
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 108059 , 2024

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

7

Effect size analysis of cipaglucosidase alfa plus miglustat..:

Mozaffar, Tahseen ; Bratkovic, Drago ; Byrne, Barry J....
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 107971 , 2024

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

8

Perspectives of the Friedreich ataxia community on gene the..:

Trantham, Shandra J. ; Coker, Mackenzi A. ; Norman, Samantha...
Molecular Therapy - Methods & Clinical Development. 32 (2024) 1 - p. 101179 , 2024

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

9

Thrombotic microangiopathy following systemic AAV administr..:

Salabarria, Stephanie M. ; Corti, Manuela ; Coleman, Kirsten E....
Journal of Clinical Investigation. 134 (2024) 1 - p. , 2024

Link: https://doi.org/10.1172/..

10

104-week efficacy and safety of cipaglucosidase alfa plus m..:

Schoser, Benedikt ; Kishnani, Priya S. ; Bratkovic, Drago...
Journal of Neurology. 271 (2024) 5 - p. 2810-2823 , 2024

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

11

P028: Switching treatment to cipaglucosidase alfa+miglustat..:

Kishnani, Priya ; Byrne, Barry ; Claeys, Kristl...
Genetics in Medicine Open. 2 (2024) - p. 100905 , 2024

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

12

B cell focused transient immune suppression protocol for ef..:

Rana, Jyoti ; Herzog, Roland W. ; Muñoz-Melero, Maite...
Molecular Therapy - Methods & Clinical Development. 32 (2024) 1 - p. 101216 , 2024

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

13

Longitudinal changes of swallowing safety and efficiency in..:

Leon‐Astudillo, Carmen ; Brooks, Olivia ; Salabarria, Stephanie M....
Pediatric Pulmonology. 59 (2024) 5 - p. 1364-1371 , 2024

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

14

Findings from the Longitudinal CINRG Becker Natural History..:

Clemens, Paula R. ; Gordish-Dressman, Heather ; Niizawa, Gabriela...
Journal of Neuromuscular Diseases. 11 (2024) 1 - p. 201-212 , 2024

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

15

Polysomnography findings in children with spinal muscular a..:

Leon-Astudillo, Carmen ; Wagner, Mary ; Salabarria, Stephanie M....
Sleep Medicine. 101 (2023) - p. 234-237 , 2023

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