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Mozaffar, Tahseen
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1

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/..
 
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2

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/..
 
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3

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/..
 
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4

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/..
 
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5

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/..
 
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6

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/..
 
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7

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/..
 
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8

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/..
 
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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/..
 
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10

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/..
 
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11

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

Byrne, Barry J. ; Schoser, Benedikt ; Kishnani, Priya...
Molecular Genetics and Metabolism.  138 (2023)  2 - p. 107042 , 2023
Link: https://doi.org/10.1016/..
 
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12

Misdiagnosis of Late-Onset Pompe Disease: A Case Series (P6..:

Huang, Dennis ; Mozaffar, Tahseen
Neurology.  100 (2023)  17_supplement_2 - p. , 2023
Link: https://doi.org/10.1212/..
 
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13

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/..
 
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14

Twelve-month functional change in Limb Girdle Muscular Dyst..:

Johnson, Nicholas ; Bates, Kameron ; Xie, Han...
Neurology.  100 (2023)  17_supplement_2 - p. , 2023
Link: https://doi.org/10.1212/..
 
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15

Safety and outcomes with efgartigimod use for acetylcholine..:

Katyal, Nakul ; Halldorsdottir, Karen ; Govindarajan, Raghav...
Muscle & Nerve.  68 (2023)  5 - p. 762-766 , 2023
Link: https://doi.org/10.1002/..
 
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