Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II
Catalano, Fabio, Vlaar, Eva C., Katsavelis, Drosos, Dammou, Zina, Huizer, Tessa F., van den Bosch, Jeroen C., Hoogeveen-Westerveld, Marianne, van den Hout, Hannerieke J.M.P., Oussoren, Esmeralda, Ruijter, George J.G., Schaaf, Gerben, Pike-Overzet, Karin, Staal, Frank J.T., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim
Published in Molecular therapy. Methods & clinical development (14.12.2023)
Published in Molecular therapy. Methods & clinical development (14.12.2023)
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A Generic Assay to Detect Aberrant ARSB Splicing and mRNA Degradation for the Molecular Diagnosis of MPS VI
Broeders, Mike, Smits, Kasper, Goynuk, Busra, Oussoren, Esmee, van den Hout, Hannerieke J.M.P., Bergsma, Atze J., van der Ploeg, Ans T., Pijnappel, W.W.M. Pim
Published in Molecular therapy. Methods & clinical development (11.12.2020)
Published in Molecular therapy. Methods & clinical development (11.12.2020)
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Effects of enzyme replacement therapy on cardiac function in classic infantile Pompe disease
Scheffers, L.E., Kok, R., van den Berg, L.E., van den Hout, J.M.P., Boersma, E., van Capelle, C.I., Helbing, W.A., van der Ploeg, A.T., Koopman, L.P.
Published in International journal of cardiology (01.06.2023)
Published in International journal of cardiology (01.06.2023)
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Cognitive outcome of patients with classic infantile Pompe disease receiving enzyme therapy
Ebbink, B J, Aarsen, F K, van Gelder, C M, van den Hout, J M P, Weisglas-Kuperus, N, Jaeken, J, Lequin, M H, Arts, W F M, van der Ploeg, A T
Published in Neurology (08.05.2012)
Published in Neurology (08.05.2012)
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Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage
Catalano, Fabio, Vlaar, Eva C, Dammou, Zina, Katsavelis, Drosos, Huizer, Tessa F, Zundo, Giacomo, Hoogeveen-Westerveld, Marianne, Oussoren, Esmeralda, van den Hout, Hannerieke J M P, Schaaf, Gerben, Pike-Overzet, Karin, Staal, Frank J T, van der Ploeg, Ans T, Pijnappel, W W M Pim
Published in Human gene therapy (01.04.2024)
Published in Human gene therapy (01.04.2024)
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A genetic modifier of symptom onset in Pompe diseaseResearch in context
Atze J. Bergsma, Stijn L.M. in 't Groen, Jan J.A. van den Dorpel, Hannerieke J.M.P. van den Hout, Nadine A.M.E. van der Beek, Benedikt Schoser, Antonio Toscano, Olimpia Musumeci, Bruno Bembi, Andrea Dardis, Amelia Morrone, Albina Tummolo, Elisabetta Pasquini, Ans T. van der Ploeg, W.W.M. Pim Pijnappel
Published in EBioMedicine (01.05.2019)
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Published in EBioMedicine (01.05.2019)
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Broad variation in phenotypes for common GAA genotypes in Pompe disease
Niño, Monica Y., in't Groen, Stijn L. M., Faria, Douglas O. S., Hoogeveen‐Westerveld, Marianne, Hout, Hannerieke J. M. P., Ploeg, Ans T., Bergsma, Atze J., Pijnappel, W. W. M. Pim
Published in Human mutation (01.11.2021)
Published in Human mutation (01.11.2021)
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A genetic modifier of symptom onset in Pompe disease
Bergsma, Atze J., in 't Groen, Stijn L.M., van den Dorpel, Jan J.A., van den Hout, Hannerieke J.M.P., van der Beek, Nadine A.M.E., Schoser, Benedikt, Toscano, Antonio, Musumeci, Olimpia, Bembi, Bruno, Dardis, Andrea, Morrone, Amelia, Tummolo, Albina, Pasquini, Elisabetta, van der Ploeg, Ans T., Pijnappel, W.W.M. Pim
Published in EBioMedicine (01.05.2019)
Published in EBioMedicine (01.05.2019)
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Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy
Winkel, Léon P. F., Kamphoven, Joep H. J., Van Den Hout, Hannerieke J. M. P., Severijnen, Lies A., Van Doorn, Pieter A., Reuser, Arnold J. J., Van Der Ploeg, Ans T.
Published in Muscle & nerve (01.06.2003)
Published in Muscle & nerve (01.06.2003)
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