E-LIB Suche - Ergebnisse für: Mozaffar, Tahseen

1

Recurring homozygous ACTN2 variant (p.Arg506Gly) causes a r..:

Donkervoort, Sandra ; Mohassel, Payam ; O'Leary, Melanie...
Annals of Clinical and Translational Neurology. 11 (2024) 3 - p. 629-640 , 2024

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

2

Case study of CD19 CAR T therapy in a subject with immune-m..:

Volkov, Jenell ; Nunez, Daniel ; Mozaffar, Tahseen...
Molecular Therapy. , 2024

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

3

Defining clinical endpoints in limb girdle muscular dystrop..:

Doody, Amy ; Alfano, Lindsay ; Diaz-Manera, Jordi...
BMC Neurology. 24 (2024) 1 - p. , 2024

Link: https://doi.org/10.1186/..

4

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/..

5

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/..

6

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/..

7

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/..

8

A Phase 1/2 first-in-human study to evaluate the safety, to..:

Rodriguez-Buritica, David F. ; Mozaffar, Tahseen ; Comi, Giacomo P....
Molecular Genetics and Metabolism. 141 (2024) 4 - p. 108250 , 2024

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

9

Debamestrocel multimodal effects on biomarker pathways in a..:

Lindborg, Stacy R. ; Goyal, Namita A. ; Katz, Jonathan...
Muscle & Nerve. 69 (2024) 6 - p. 719-729 , 2024

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

10

A study to evaluate seroprevalence of antibodies to AAV8 an..:

Walzer, Mark ; Shah, Ankita ; Malayang, Jill...
Molecular Genetics and Metabolism. 141 (2024) 2 - p. 108068 , 2024

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

11

Efficacy of avalglucosidase alfa on forced vital capacity p..:

Mozaffar, Tahseen ; Riou França, Lionel ; Msihid, Jérôme...
Molecular Genetics and Metabolism Reports. 40 (2024) - p. 101109 , 2024

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

12

272nd ENMC international workshop: 10 Years of progress - r..:

Lilleker, James B. ; Naddaf, Elie ; Saris, Christiaan G.J....
Neuromuscular Disorders. 37 (2024) - p. 36-51 , 2024

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

13

Change in Concomitant Therapies for Generalized Myasthenia ..:

Pulley, Michael ; Macwan, Samir ; Nowak, Richard...
Neurology. 100 (2023) 17_supplement_2 - p. , 2023

Link: https://doi.org/10.1212/..

14

P021: Long-term follow-up of cipaglucosidase alfa/miglustat..:

Kishnani, Priya ; Schoser, Benedikt ; Bratkovic, Drago...
Genetics in Medicine Open. 1 (2023) 1 - p. 100031 , 2023

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

15

AT845 gene replacement therapy for late onset Pompe disease..:

Manera, Jordi Diaz ; Mozaffar, Tahseen ; Day, John W....
Molecular Genetics and Metabolism. 138 (2023) 2 - p. 107078 , 2023

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