E-LIB Suche - Ergebnisse für: Byrne, Barry

1

Emerging therapies in hereditary ataxias:

Eisel, Mallory L.S. ; Burns, Matthew ; Ashizawa, Tetsuo...
Trends in Molecular Medicine. , 2024

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

2

Neurological glycogen storage diseases and emerging therape..:

Colpaert, Matthieu ; Singh, Pankaj K. ; Donohue, Katherine J....
Neurotherapeutics. , 2024

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

3

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/..

4

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/..

5

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/..

6

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/..

7

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/..

8

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/..

9

Meeting Report: 2023 Muscular Dystrophy Association Summit ..:

Lek, Angela ; Atas, Evrim ; Lin, Brian...
Journal of Neuromuscular Diseases. 11 (2024) 5 - p. 1139-1160 , 2024

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

10

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/..

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

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/..

13

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/..

14

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/..

15

Codon-Optimized and de novo–Synthesized E-Selectin/AAV2 Dos..:

Voza, Francesca A. ; Byrne, Barry J. ; Ortiz, Yulexi Y....
Annals of Surgery. 280 (2024) 4 - p. 570-583 , 2024

Link: https://doi.org/10.1097/..