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Switching Enzyme Replacement Therapy for Late‐Onset Pompe Disease From Alglucosidase Alfa to Cipaglucosidase Alfa Plus Miglustat: Post Hoc Effect Size Analysis of PROPEL
Kushlaf, Hani, Díaz‐Manera, Jordi, Bratkovic, Drago, Byrne, Barry J., Claeys, Kristl G., Clemens, Paula R., Dimachkie, Mazen M., Kishnani, Priya S., Laforêt, Pascal, Roberts, Mark, Schoser, Benedikt, Toscano, Antonio, Castelli, Jeff, Holdbrook, Fred, Sitaraman Das, Sheela, Goldman, Mitchell, Mozaffar, Tahseen
Published in Muscle & nerve (01.08.2025)
Published in Muscle & nerve (01.08.2025)
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Journal Article
Safety and effectiveness of resistance training in patients with late onset Pompe disease - a pilot study
Bhatnagar, Caleb, Shah, Jeet, Ramani, Bhumi, Surampalli, Abhilasha, Avanti, Mahima, Radom-Aizik, Shlomit, Knight, Margaret, Weiss, Lan, Caiozzo, Vince, Kimonis, Virginia
Published in Neuromuscular disorders : NMD (01.04.2022)
Published in Neuromuscular disorders : NMD (01.04.2022)
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Journal Article
Analysis of the Italian cohort of late-onset Pompe disease (LOPD) patients after 10 and 15 years of therapy with alglucosidase alfa
Mongini, T., Gadaleta, G., Alonge, P., Vercelli, L., Stura, I., Musumeci, O., Ravaglia, S., Ruggiero, L., Fiumara, A., Barone, R., Servidei, S., Sancricca, C., Siciliano, G., Ricci, G., Sechi, A., Tonin, P., Pegoraro, E., Filosto, M., D’Angelo, G., Comi, G., Maggi, L., Barp, A., Crescimanno, G., Toscano, A.
Published in Journal of neurology (11.07.2025)
Published in Journal of neurology (11.07.2025)
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Journal Article
Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity
Smith, Barbara K., Martin, A. Daniel, Lawson, Lee Ann, Vernot, Valerie, Marcus, Jordan, Islam, Saleem, Shafi, Nadeem, Corti, Manuela, Collins, Shelley W., Byrne, Barry J.
Published in Experimental neurology (01.01.2017)
Published in Experimental neurology (01.01.2017)
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Journal Article
A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease
Byrne, Barry J., Geberhiwot, Tarekegn, Barshop, Bruce A., Barohn, Richard, Hughes, Derralynn, Bratkovic, Drago, Desnuelle, Claude, Laforet, Pascal, Mengel, Eugen, Roberts, Mark, Haroldsen, Peter, Reilley, Kristin, Jayaram, Kala, Yang, Ke, Walsh, Liron
Published in Orphanet journal of rare diseases (24.08.2017)
Published in Orphanet journal of rare diseases (24.08.2017)
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Journal Article
B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Disease
Elder, Melissa E., Nayak, Sushrusha, Collins, Shelley W., Lawson, Lee Ann, Kelley, Jeffry S., Herzog, Roland W., Modica, Renee F., Lew, Judy, Lawrence, Robert M., Byrne, Barry J.
Published in The Journal of pediatrics (01.09.2013)
Published in The Journal of pediatrics (01.09.2013)
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Journal Article
Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review
Ditters, Imke A. M., van Kooten, Harmke A., van der Beek, Nadine A. M. E., van der Ploeg, Ans T., Huidekoper, Hidde H., van den Hout, Johanna M. P.
Published in Biomolecules (Basel, Switzerland) (01.09.2023)
Published in Biomolecules (Basel, Switzerland) (01.09.2023)
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Journal Article
Switching treatment to cipaglucosidase alfa plus miglustat positively affects patient-reported outcome measures in patients with late-onset Pompe disease
Kishnani, Priya S., Byrne, Barry J., Claeys, Kristl G., Díaz-Manera, Jordi, Dimachkie, Mazen M., Kushlaf, Hani, Mozaffar, Tahseen, Roberts, Mark, Schoser, Benedikt, Hummel, Noemi, Kopiec, Agnieszka, Holdbrook, Fred, Shohet, Simon, Toscano, Antonio
Published in Journal of patient-reported outcomes (13.11.2024)
Published in Journal of patient-reported outcomes (13.11.2024)
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Journal Article
Oropharyngeal Dysphagia in Infants and Children with Infantile Pompe Disease
Jones, Harrison N., Muller, Carolyn W., Lin, Min, Banugaria, Suhrad G., Case, Laura E., Li, Jennifer S., O’Grady, Gwendolyn, Heller, James H., Kishnani, Priya S.
Published in Dysphagia (01.12.2010)
Published in Dysphagia (01.12.2010)
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Journal Article
Increased Risk of Gastrointestinal Malignancy in Patients with Diabetes Mellitus and Correlations with Anti-Diabetes Drugs: A Nationwide Population-based Study in Taiwan
Liang, Ying, Chan, Wan-Leong, Chen, Jaw-Wen, Chen, Yu-Chun, Chiu, Chun-Chih, Chen, Tzeng-Ji, Huang, Chin-Chou, Lin, Shing-Jong, Leu, Hsin-Bang
Published in Internal Medicine (01.01.2013)
Published in Internal Medicine (01.01.2013)
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Journal Article
An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions
El-Gharbawy, Areeg H., Mackey, Joanne, DeArmey, Stephanie, Westby, Greg, Grinnell, Sherry G., Malovrh, Peggy, Conway, Robert, Kishnani, Priya S.
Published in Molecular genetics and metabolism (01.09.2011)
Published in Molecular genetics and metabolism (01.09.2011)
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Journal Article
Early cognitive development in children with infantile Pompe disease
Spiridigliozzi, Gail A., Heller, James H., Case, Laura E., Jones, Harrison N., Kishnani, Priya S.
Published in Molecular genetics and metabolism (01.03.2012)
Published in Molecular genetics and metabolism (01.03.2012)
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Journal Article
Enzyme Replacement Therapy Improves Respiratory Outcomes in Patients with Late-Onset Type II Glycogenosis and High Ventilator Dependency
Vianello, Andrea, Semplicini, Claudio, Paladini, Luciana, Concas, Alessandra, Ravaglia, Sabrina, Servidei, Serenella, Toscano, Antonio, Mongini, Tiziana, Angelini, Corrado, Pegoraro, Elena
Published in Lung (01.10.2013)
Published in Lung (01.10.2013)
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Journal Article
Use of cardiac magnetic resonance imaging to evaluate cardiac structure, function and fibrosis in children with infantile Pompe disease on enzyme replacement therapy
Barker, Piers C.A., Pasquali, Sara K., Darty, Stephen, Ing, Richard J., Li, Jennifer S., Kim, Raymond J., DeArmey, Stephanie, Kishnani, Priya S., Campbell, Michael J.
Published in Molecular genetics and metabolism (01.12.2010)
Published in Molecular genetics and metabolism (01.12.2010)
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Journal Article