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Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease by Barry J. Byrne, Giancarlo Parenti, Giancarlo Parenti, Benedikt Schoser, Ans T. van der Ploeg, Hung Do, Brian Fox, Mitchell Goldman, Franklin K. Johnson, Jia Kang, Nickita Mehta, John Mondick, M. Osman Sheikh, Sheela Sitaraman Das, Steven Tuske, Jon Brudvig, Jill M. Weimer, Tahseen Mozaffar
Published 2025-01-01Get full text
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Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease by Barry J. Byrne, Giancarlo Parenti, Giancarlo Parenti, Benedikt Schoser, Ans T. van der Ploeg, Hung Do, Brian Fox, Mitchell Goldman, Franklin K. Johnson, Jia Kang, Nickita Mehta, John Mondick, M. Osman Sheikh, Sheela Sitaraman Das, Steven Tuske, Jon Brudvig, Jill M. Weimer, Tahseen Mozaffar
Published 2024-10-01Get full text
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Clinical Trial Readiness in Limb Girdle Muscular Dystrophy R1 (LGMDR1): A GRASP Consortium Study by Stephanie M. Hunn, Lindsay N. Alfano, Aileen Jones, Amanda Butler, Linda P. Lowes, Megan A. Iammarino, Natalie F. Reash, Lindsay Pietruszewski, Sandhya Sasidharan, Melissa Currence, Jeffrey M. Statland, Talia Strahler, Robert Will, Matthew Wicklund, Stacy Dixon, Renee Augsburger, Tahseen Mozaffar, Katie M. Laubscher, Shelley R. H. Mockler, Katherine D. Mathews, Nikia Stinson, Doris G. Leung, Molly M. Stark, Rebecca A. Horton, Peter B. Kang, Meredith K. James, Amanda Clause, Conrad C. Weihl, Nicholas E. Johnson, GRASP‐LGMD Consortium
Published 2025-06-01Get full text
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Prospective observational study of FKRP‐related limb‐girdle muscular dystrophy R9: A GRASP consortium study by Lindsay N. Alfano, Meredith K. James, Kristine Grosfjeld Petersen, Karen Rudolf, John Vissing, Renee Augsburger, Tahseen Mozaffar, Aileen Jones, Amanda Butler, Katie M. Laubscher, Shelley R. H. Mockler, Katherine D. Mathews, Megan A. Iammarino, Natalie F. Reash, Lindsay Pietruszewski, Linda P. Lowes, Talia Strahler, Matthew Wicklund, Stephanie Hunn, Conrad C. Weihl, Sandhya Sasidharan, Melissa Currence, Jeffrey M. Statland, Nikia Stinson, Megan Holzer, Doris G. Leung, Donovan J. Lott, Peter B. Kang, Scott Holsten, Urvi Desai, Nicholas E. Johnson, the GRASP‐LGMD Consortium
Published 2025-02-01Get full text
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