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
Home-based enzyme replacement therapy in children and adults with Pompe disease; a prospective study
Ditters, Imke A M, van der Beek, Nadine A M E, Brusse, Esther, van der Ploeg, Ans T, van den Hout, Johanna M P, Huidekoper, Hidde H
Published in Orphanet journal of rare diseases (08.05.2023)
Published in Orphanet journal of rare diseases (08.05.2023)
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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
Pain: a prevalent feature in patients with mucopolysaccharidosis. Results of a cross-sectional national survey
Brands, Marion M. G., Güngör, Deniz, van den Hout, Johanna M. P., Karstens, Francois P. J., Oussoren, Esmee, Plug, Iris, Boelens, Jaap Jan, van Hasselt, Peter M., Hollak, Carla E. M., Mulder, Margot F., Gozalbo, Estela Rubio, Smeitink, Jan A., Smit, G. Peter A., Wijburg, Frits A., Meutgeert, Hanka, van der Ploeg, Ans T.
Published in Journal of inherited metabolic disease (01.03.2015)
Published in Journal of inherited metabolic disease (01.03.2015)
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Journal Article
Long-term cognitive follow-up in children treated for Maroteaux-Lamy syndrome
Ebbink, Berendine J., Brands, Marion M. G., van den Hout, Johanna M. P., Lequin, Maarten H., van den Braak, Robert R. J. Coebergh, van de Weitgraven, Rianne L., Plug, Iris, Aarsen, Femke K., van der Ploeg, Ans T.
Published in Journal of inherited metabolic disease (01.03.2016)
Published in Journal of inherited metabolic disease (01.03.2016)
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Journal Article
Classic infantile Pompe patients approaching adulthood: a cohort study on consequences for the brain
Ebbink, Berendine J, Poelman, Esther, Aarsen, Femke K, Plug, Iris, Régal, Luc, Muentjes, Carsten, Beek, Nadine A M E, Lequin, Maarten H, Ploeg, Ans T, Hout, Johanna M P
Published in Developmental medicine and child neurology (01.06.2018)
Published in Developmental medicine and child neurology (01.06.2018)
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Journal Article
Analysis of urinary oligosaccharide excretion patterns by UHPLC/HRAM mass spectrometry for screening of lysosomal storage disorders
Hagemeijer, Marne C., Bosch, Jeroen C., Bongaerts, Michiel, Jacobs, Edwin H., Hout, Johanna M. P., Oussoren, Esmee, Ruijter, George J. G.
Published in Journal of inherited metabolic disease (01.03.2023)
Published in Journal of inherited metabolic disease (01.03.2023)
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Journal Article
Diffusion tensor imaging of the brain in Pompe disease
van den Dorpel, Jan J. A., Dremmen, Marjolein H. G., van der Beek, Nadine A. M. E., Rizopoulos, Dimitris, van Doorn, Pieter A., van der Ploeg, Ans T., Muetzel, Ryan L., van den Hout, Johanna M. P.
Published in Journal of neurology (01.03.2023)
Published in Journal of neurology (01.03.2023)
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Journal Article
Start, switch and stop (triple‐S) criteria for enzyme replacement therapy of late‐onset Pompe disease: European Pompe Consortium recommendation update 2024
Schoser, Benedikt, Beek, Nadine A. M. E., Broomfield, Alexander, Brusse, Esther, Diaz‐Manera, Jordi, Hahn, Andreas, Hundsberger, Thomas, Kornblum, Cornelia, Kruijshaar, Michelle, Laforet, Pascal, Mengel, Eugen, Mongini, Tiziana, Orlikowski, David, Parenti, Giancarlo, Pijnappel, W. W. M. Pim, Roberts, Mark, Scherer, Thomas, Toscano, Antonio, Vissing, John, Hout, Johanna M. P., Doorn, Pieter A., Wenninger, Stephan, Ploeg, Ans T.
Published in European journal of neurology (01.09.2024)
Published in European journal of neurology (01.09.2024)
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Journal Article
Effects of higher and more frequent dosing of alglucosidase alfa and immunomodulation on long‐term clinical outcome of classic infantile Pompe patients
Poelman, Esther, Dorpel, Jan J. A., Hoogeveen‐Westerveld, Marianne, Hout, Johanna M. P., Giessen, Lianne J., Beek, Nadine A. M. E., Pijnappel, W. W. M. Pim, Ploeg, Ans T.
Published in Journal of inherited metabolic disease (01.11.2020)
Published in Journal of inherited metabolic disease (01.11.2020)
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Journal Article
Neurofilament light protein as a biomarker for spinal muscular atrophy: a review and reference ranges
Bayoumy, Sherif, Verberk, Inge M.W., Vermunt, Lisa, Willemse, Eline, den Dulk, Ben, van der Ploeg, Ans T., Pajkrt, Dasja, Nitz, Elisa, van den Hout, Johanna M.P., van der Post, Julie, Wolf, Nicole I., Beerepoot, Shanice, Groen, Ewout J.N., Tüngler, Victoria, Teunissen, Charlotte E.
Published in Clinical chemistry and laboratory medicine (25.06.2024)
Published in Clinical chemistry and laboratory medicine (25.06.2024)
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Journal Article
Can serial cerebral MRIs predict the neuronopathic phenotype of MPS II?
Vollebregt, Audrey A. M., Ebbink, Berendine J., Rizopoulos, Dimitris, Lequin, Maarten H., Aarsen, Femke K., Shapiro, Elsa G., Ploeg, Ans T., Hout, Johanna M. P.
Published in Journal of inherited metabolic disease (01.05.2021)
Published in Journal of inherited metabolic disease (01.05.2021)
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Journal Article
Cognitive decline in classic infantile Pompe disease: An underacknowledged challenge
Ebbink, Berendine J, Poelman, Esther, Plug, Iris, Lequin, Maarten H, van Doorn, Pieter A, Aarsen, Femke K, van der Ploeg, Ans T, van den Hout, Johanna M P
Published in Neurology (29.03.2016)
Published in Neurology (29.03.2016)
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Journal Article
Is the brain involved in patients with late‐onset Pompe disease?
Dorpel, Jan J. A., Vlugt, Willemijn M. C., Dremmen, Marjolein H. G., Muetzel, Ryan, Berg, Esther, Hest, Roos, Kriek, Joni, Brusse, Esther, Doorn, Pieter A., Ploeg, Ans T., Hout, Johanna M. P., Beek, Nadine A. M. E.
Published in Journal of inherited metabolic disease (01.05.2022)
Published in Journal of inherited metabolic disease (01.05.2022)
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Journal Article
Craniosynostosis affects the majority of mucopolysaccharidosis patients and can contribute to increased intracranial pressure
Oussoren, Esmee, Mathijssen, Irene M. J., Wagenmakers, Margreet, Verdijk, Rob M., Bredero-Boelhouwer, Hansje H., van Veelen-Vincent, Marie-Lise C., van der Meijden, Jan C., van den Hout, Johanna M. P., Ruijter, George J. G., van der Ploeg, Ans T., Langeveld, Mirjam
Published in Journal of inherited metabolic disease (01.12.2018)
Published in Journal of inherited metabolic disease (01.12.2018)
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Journal Article
Enzyme replacement therapy in late-onset Pompe's disease: A three-year follow-up
Winkel, Léon P. F., Van den Hout, Johanna M. P., Kamphoven, Joep H. J., Disseldorp, Janus A. M., Remmerswaal, Maaike, Arts, Willem F. M., Loonen, M. Christa B., Vulto, Arnold G., Van Doorn, Pieter A., De Jong, Gerard, Hop, Wim, Smit, G. Peter A., Shapira, Stuart K., Boer, Marijke A., Reuser, Arnold J. J., Van der Ploeg, Ans T., van Diggelen, Otto P.
Published in Annals of neurology (01.04.2004)
Published in Annals of neurology (01.04.2004)
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Journal Article
Neurofilament Light and Its Association With CNS Involvement in Patients With Classic Infantile Pompe Disease
Mackenbach, Maarten J, Willemse, Eline A J, van den Dorpel, Jan J A, van der Beek, Nadine A M E, Díaz-Manera, Jordi, Rizopoulos, Dimitris, Teunissen, Charlotte, van der Ploeg, Ans T, van den Hout, Johanna M P
Published in Neurology (08.08.2023)
Published in Neurology (08.08.2023)
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Journal Article
Cardiac outcome in classic infantile Pompe disease after 13 years of treatment with recombinant human acid alpha-glucosidase
van Capelle, Carine I., Poelman, Esther, Frohn-Mulder, Ingrid M., Koopman, Laurens P., van den Hout, Johanna M.P., Régal, Luc, Cools, Bjorn, Helbing, Wim A., van der Ploeg, Ans T.
Published in International journal of cardiology (15.10.2018)
Published in International journal of cardiology (15.10.2018)
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Journal Article
High Sustained Antibody Titers in Patients with Classic Infantile Pompe Disease Following Immunomodulation at Start of Enzyme Replacement Therapy
Poelman, Esther, Hoogeveen-Westerveld, Marianne, Kroos-de Haan, Marian A., van den Hout, Johanna M.P., Bronsema, Kees J., van de Merbel, Nico C., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim
Published in The Journal of pediatrics (01.04.2018)
Published in The Journal of pediatrics (01.04.2018)
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Journal Article
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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