Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions
Ditters, Imke A. M., van Kooten, Harmke A., van der Beek, Nadine A. M. E., Hardon, Jacqueline F., Ismailova, Gamida, Brusse, Esther, Kruijshaar, Michelle E., van der Ploeg, Ans T., van den Hout, Johanna M. P., Huidekoper, Hidde H.
Published in BioDrugs : clinical immunotherapeutics, biopharmaceuticals, and gene therapy (01.09.2023)
Published in BioDrugs : clinical immunotherapeutics, biopharmaceuticals, and gene therapy (01.09.2023)
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Journal Article
Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease
van Kooten, Harmke A, Ditters, Imke A M, Hoogeveen-Westerveld, Marianne, Jacobs, Edwin H, van den Hout, Johanna M P, van Doorn, Pieter A, Pijnappel, W W M Pim, van der Ploeg, Ans T, van der Beek, Nadine A M E
Published in Orphanet journal of rare diseases (02.02.2022)
Published in Orphanet journal of rare diseases (02.02.2022)
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Journal Article
Chest MRI to diagnose early diaphragmatic weakness in Pompe disease
Harlaar, Laurike, Ciet, Pierluigi, van Tulder, Gijs, Pittaro, Alice, van Kooten, Harmke A, van der Beek, Nadine A M E, Brusse, Esther, Wielopolski, Piotr A, de Bruijne, Marleen, van der Ploeg, Ans T, Tiddens, Harm A W M, van Doorn, Pieter A
Published in Orphanet journal of rare diseases (07.01.2021)
Published in Orphanet journal of rare diseases (07.01.2021)
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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
MRI changes in diaphragmatic motion and curvature in Pompe disease over time
Harlaar, Laurike, Ciet, Pierluigi, van Tulder, Gijs, van Kooten, Harmke A., van der Beek, Nadine A. M. E., Brusse, Esther, de Bruijne, Marleen, Tiddens, Harm A. W. M., van der Ploeg, Ans T., van Doorn, Pieter A.
Published in European radiology (01.12.2022)
Published in European radiology (01.12.2022)
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Journal Article
Improving outcome measures in late onset Pompe disease: Modified Rasch‐Built Pompe‐Specific Activity scale
van Kooten, Harmke A., Horton, Mike C., Wenninger, Stephan, Babačić, Haris, Schoser, Benedikt, Lefeuvre, Claire, Taouagh, Najib, Laforêt, Pascal, Segovia, Sonia, Díaz‐Manera, Jordi, Claeys, Kristl G., Mongini, Tiziana, Musumeci, Olimpia, Toscano, Antonio, Hundsberger, Thomas, Brusse, Esther, van Doorn, Pieter A., van der Ploeg, Ans T., van der Beek, Nadine A. M. E.
Published in European journal of neurology (28.08.2024)
Published in European journal of neurology (28.08.2024)
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