I agree that this site is using cookies. You can find further informations here.
X
Dimachkie, Mazen M.
~ 300  results:
Search for persons X
?
1

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/..
 
?
2

Pattern Recognition of Neuropathy and Neuronopathy: 7 Quest..:

Barohn, Richard J. ; Dimachkie, Mazen M. ; Levine, Todd D...
RRNMF Neuromuscular Journal.  5 (2024)  1 - p. , 2024
Link: https://doi.org/10.17161..
 
?
3

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/..
 
?
4

COMET post hoc analysis: Efficacy of long-term avalglucosid..:

Toscano, Antonio ; Kishnani, Priya ; Dimachkie, Mazen M....
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 108058 , 2024
Link: https://doi.org/10.1016/..
 
?
5

Safety and tolerability of intravenous immunoglobulin in pa..:

Aggarwal, Rohit ; Schessl, Joachim ; Charles-Schoeman, Christina...
Arthritis Research & Therapy.  26 (2024)  1 - p. , 2024
Link: https://doi.org/10.1186/..
 
?
6

Applying the win ratio method in clinical trials of orphan ..:

Boentert, Matthias ; Berger, Kenneth I. ; Díaz-Manera, Jordi...
Orphanet Journal of Rare Diseases.  19 (2024)  1 - p. , 2024
Link: https://doi.org/10.1186/..
 
?
7

Post-hoc Nonparametric Analysis of Forced Vital Capacity in..:

Boentert, Matthias ; Campana, Emmanuelle Salort ; Attarian, Shahram...
Journal of Neuromuscular Diseases.  11 (2024)  2 - p. 369-374 , 2024
Link: https://doi.org/10.3233/..
 
?
8

Effect of avalglucosidase alfa on disease-specific and gene..:

Toscano, Antonio ; Pollissard, Laurence ; Msihid, Jérôme...
Molecular Genetics and Metabolism.  141 (2024)  2 - p. 108121 , 2024
Link: https://doi.org/10.1016/..
 
?
9

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/..
 
?
10

Avalglucosidase alfa improves motor and respiratory functio..:

Diaz-Manera, Jordi ; Dimachkie, Mazen M. ; Ishak, Jack...
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107076 , 2023
Link: https://doi.org/10.1016/..
 
?
11

Safety and efficacy of arimoclomol for inclusion body myosi..:

Machado, Pedro M ; McDermott, Michael P ; Blaettler, Thomas...
The Lancet Neurology.  22 (2023)  10 - p. 900-911 , 2023
Link: https://doi.org/10.1016/..
 
?
12

Measurement Properties of 2 Novel PROs, the Pompe Disease S..:

Dimachkie, Mazen M. ; Kishnani, Priya S. ; Ivanescu, Cristina...
Neurology Clinical Practice.  13 (2023)  5 - p. , 2023
Link: https://doi.org/10.1212/..
 
?
13

Moving forward together: collaborative landscapes of resear..:

Saketkoo, Lesley Ann ; Valenzuela, Antonia ; Kim, Susan...
Rheumatology.  63 (2023)  5 - p. 1189-1191 , 2023
Link: https://doi.org/10.1093/..
 
?
14

Phase 2 trial in acetylcholine receptor antibody‐positive m..:

Pasnoor, Mamatha ; Bril, Vera ; Levine, Todd...
European Journal of Neurology.  30 (2023)  5 - p. 1417-1424 , 2023
Link: https://doi.org/10.1111/..
 
?
15

Plasma proteomic analysis on neuropathic pain in idiopathic..:

van Doormaal, Perry T. C. ; Thomas, Simone ; Ajroud‐Driss, Senda...
Journal of the Peripheral Nervous System.  29 (2023)  1 - p. 88-96 , 2023
Link: https://doi.org/10.1111/..
 
1-15