Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease von Byrne, Barry J, Parenti, Giancarlo, Schoser, Benedikt, van der Ploeg, Ans T, Do, Hung, Fox, Brian, Goldman, Mitchell, Johnson, Franklin K, Kang, Jia, Mehta, Nickita, Mondick, John, Sheikh, M Osman, Sitaraman Das, Sheela, Tuske, Steven, Brudvig, Jon, Weimer, Jill M, Mozaffar, Tahseen

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Validation of the Patient-Reported Outcomes Measurement Information System (PROMIS®) physical function questionnaire in late-onset Pompe disease using PROPEL phase 3 data von Kishnani, Priya S., Shohet, Simon, Raza, Syed, Hummel, Noemi, Castelli, Jeffrey P., Sitaraman Das, Sheela, Jiang, Heng, Kopiec, Agnieszka, Keyzor, Ian, Hahn, Andreas

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P021: Long-term follow-up of cipaglucosidase alfa/miglustat in ambulatory and non-ambulatory patients with Pompe disease: An open-label phase I/II study (ATB200-02) von Kishnani, Priya, Schoser, Benedikt, Bratkovic, Drago, Clemens, Paula, Goker-Alpan, Ozlem, Ming, Xue, Roberts, Mark, Vorgerd, Matthias, Sivakumar, Kumaraswamy, van der Ploeg, Ans, Goldman, Mitchell, Wright, Jacquelyn, Holdbrook, Fred, Jain, Vipul, Das, Sheela Sitaraman, Wasfi, Yasmine, Mozaffar, Tahseen, Byrne, Barry

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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07) von Schoser, Benedikt, Kishnani, Priya S, Bratkovic, Drago, Byrne, Barry J, Claeys, Kristl G, Diaz-Manera, Jordi, Laforet, Pascal, Roberts, Mark, Toscano, Antonio, van der Ploeg, Ans T, Castelli, Jeff, Goldman, Mitchell, Holdbrook, Fred, Sitaraman Das, Sheela, Wasfi, Yasmine, Mozaffar, Tahseen

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Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease: an open-label phase I/II study (ATB200-02) von Byrne, Barry J., Schoser, Benedikt, Kishnani, Priya S., Bratkovic, Drago, Clemens, Paula R., Goker-Alpan, Ozlem, Ming, Xue, Roberts, Mark, Vorgerd, Matthias, Sivakumar, Kumaraswamy, van der Ploeg, Ans T., Goldman, Mitchell, Wright, Jacquelyn, Holdbrook, Fred, Jain, Vipul, Benjamin, Elfrida R., Johnson, Franklin, Das, Sheela Sitaraman, Wasfi, Yasmine, Mozaffar, Tahseen

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Effect size analysis of cipaglucosidase alfa plus miglustat versus alglucosidase alfa in ERT-experienced adults with late-onset Pompe disease in PROPEL von Mozaffar, Tahseen, Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Diaz-Manera, Jordi, Dimachkie, Mazen M., Kishnani, Priya S., Kushlaf, Hani, Roberts, Mark E., Toscano, Antonio, Castelli, Jeffrey, Holdbrook, Fred K., Das, Sheela Sitaraman, Wasfi, Yasmine, Schoser, Benedikt

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Immunogenicity of cipaglucosidase alfa/miglustat versus alglucosidase alfa/placebo in late-onset Pompe disease (LOPD): A phase III, randomized study (PROPEL) von Chien, Yin-Hsiu, Benjamin, Elfrida, Schoser, Benedikt, Kishnani, Priya, Mozaffar, Tahseen, Díaz-Manera, Jordi, Johnson, Franklin, Das, Sheela Sitaraman, Nair, Anju, Williams, Hadis, Anderson, Eric, Mondick, John T., Sileno, Anthony

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104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07) von Schoser, Benedikt, Kishnani, Priya S., Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Díaz-Manera, Jordi, Laforêt, Pascal, Roberts, Mark, Toscano, Antonio, van der Ploeg, Ans T., Castelli, Jeff, Goldman, Mitchell, Holdbrook, Fred, Sitaraman Das, Sheela, Wasfi, Yasmine, Mozaffar, Tahseen

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FEXAMETHODS FOR TREATING INFANTILE-ONSET POMPE DISEASE IN PEDIATRIC PATIENTS von DAS, Sheela Sitaraman, BARTH, Jay, CASTELLI, Jeff

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METHODS FOR TREATING LATE ONSET POMPE DISEASE IN PEDIATRIC PATIENTS von DAS, Sheela Sitaraman, BARTH, Jay, CASTELLI, Jeff

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METHODS FOR TREATING POMPE DISEASE von DAS, Sheela Sitaraman, BARTH, Jay, CASTELLI, Jeff

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Methods for treating infantile-onset Pompe disease in pediatric patients von DAS, SHEELA SITARAMAN, CASTELLI, JEFF, BARTH, JAY

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MÉTODOS PARA TRATAR LA ENFERMEDAD DE POMPE von DAS, SHEELA SITARAMAN, CASTELLI, JEFF, BARTH, JAY

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Methods for treating Pompe disease von DAS, SHEELA SITARAMAN, CASTELLI, JEFF, BARTH, JAY

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