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11 results on '"van Hasselt, Peter"'

1. Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Author

Schoenmakers, Daphne H., Beerepoot, Shanice, van den Berg, Sibren, Adang, Laura, Bley, Annette, Boelens, Jaap-Jan, Fumagalli, Francesca, Goettsch, Wim G., Grønborg, Sabine, Groeschel, Samuel, van Hasselt, Peter M., Hollak, Carla E. M., Lindemans, Caroline, Mochel, Fanny, Mol, Peter G. M., Sevin, Caroline, Zerem, Ayelet, Schöls, Ludger, and Wolf, Nicole I.

Published
2022
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2. Synapse alterations precede neuronal damage and storage pathology in a human cerebral organoid model of CLN3-juvenile neuronal ceroid lipofuscinosis

Author

Gomez-Giro, Gemma, Arias-Fuenzalida, Jonathan, Jarazo, Javier, Zeuschner, Dagmar, Ali, Muhammad, Possemis, Nina, Bolognin, Silvia, Halder, Rashi, Jäger, Christian, Kuper, Willemijn F. E., van Hasselt, Peter M., Zaehres, Holm, del Sol, Antonio, van der Putten, Herman, Schöler, Hans R., and Schwamborn, Jens C.

Published
2019
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3. Emotional and behavioral problems, quality of life and metabolic control in NTBC-treated Tyrosinemia type 1 patients

Author

van Vliet, Kimber, van Ginkel, Willem G., Jahja, Rianne, Daly, Anne, MacDonald, Anita, De Laet, Corinne, Vara, Roshni, Rahman, Yusof, Cassiman, David, Eyskens, Francois, Timmer, Corrie, Mumford, Nicky, Bierau, Jörgen, van Hasselt, Peter M., Gissen, Paul, Goyens, Philippe J., McKiernan, Patrick J., Wilcox, Gisela, Morris, Andrew A. M., Jameson, Elisabeth A., Huijbregts, Stephan C. J., and van Spronsen, Francjan J.

Published
2019
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4. Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: results of an international consensus procedure

Author

Kuiper, Gé-Ann, Langereis, Eveline J., Breyer, Sandra, Carbone, Marco, Castelein, René M., Eastwood, Deborah M., Garin, Christophe, Guffon, Nathalie, van Hasselt, Peter M., Hensman, Pauline, Jones, Simon A., Kenis, Vladimir, Kruyt, Moyo, van der Lee, Johanna H., Mackenzie, William G., Orchard, Paul J., Oxborrow, Neil, Parini, Rossella, Robinson, Amy, Schubert Hjalmarsson, Elke, White, Klane K., and Wijburg, Frits A.

Published
2019
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5. Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Author

Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, Schoenmakers, Daphne H, Beerepoot, Shanice, van den Berg, Sibren, Adang, Laura, Bley, Annette, Boelens, Jaap-Jan, Fumagalli, Francesca, Goettsch, Wim G, Grønborg, Sabine, Groeschel, Samuel, van Hasselt, Peter M, Hollak, Carla E M, Lindemans, Caroline, Mochel, Fanny, Mol, Peter G M, Sevin, Caroline, Zerem, Ayelet, Schöls, Ludger, Wolf, Nicole I, Afd Pharmacoepi & Clinical Pharmacology, Pharmacoepidemiology and Clinical Pharmacology, Schoenmakers, Daphne H, Beerepoot, Shanice, van den Berg, Sibren, Adang, Laura, Bley, Annette, Boelens, Jaap-Jan, Fumagalli, Francesca, Goettsch, Wim G, Grønborg, Sabine, Groeschel, Samuel, van Hasselt, Peter M, Hollak, Carla E M, Lindemans, Caroline, Mochel, Fanny, Mol, Peter G M, Sevin, Caroline, Zerem, Ayelet, Schöls, Ludger, and Wolf, Nicole I

Published
2022

6. Modified Delphi procedure-based expert consensus on endpoints for an international disease registry for Metachromatic Leukodystrophy: The European Metachromatic Leukodystrophy initiative (MLDi)

Author

CTI Nierkens, Infection & Immunity, Regenerative Medicine and Stem Cells, Metabole ziekten patientenzorg, Child Health, Schoenmakers, Daphne H., Beerepoot, Shanice, van den Berg, Sibren, Adang, Laura, Bley, Annette, Boelens, Jaap Jan, Fumagalli, Francesca, Goettsch, Wim G., Grønborg, Sabine, Groeschel, Samuel, van Hasselt, Peter M., Hollak, Carla E.M., Lindemans, Caroline, Mochel, Fanny, Mol, Peter G.M., Sevin, Caroline, Zerem, Ayelet, Schöls, Ludger, Wolf, Nicole I., CTI Nierkens, Infection & Immunity, Regenerative Medicine and Stem Cells, Metabole ziekten patientenzorg, Child Health, Schoenmakers, Daphne H., Beerepoot, Shanice, van den Berg, Sibren, Adang, Laura, Bley, Annette, Boelens, Jaap Jan, Fumagalli, Francesca, Goettsch, Wim G., Grønborg, Sabine, Groeschel, Samuel, van Hasselt, Peter M., Hollak, Carla E.M., Lindemans, Caroline, Mochel, Fanny, Mol, Peter G.M., Sevin, Caroline, Zerem, Ayelet, Schöls, Ludger, and Wolf, Nicole I.

Published
2022

7. Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I : Results of an international consensus procedure

Author

Kuiper, Gé Ann, Langereis, Eveline J., Breyer, Sandra, Carbone, Marco, Castelein, René M., Eastwood, Deborah M., Garin, Christophe, Guffon, Nathalie, Van Hasselt, Peter M., Hensman, Pauline, Jones, Simon A., Kenis, Vladimir, Kruyt, Moyo, Van Der Lee, Johanna H., Mackenzie, William G., Orchard, Paul J., Oxborrow, Neil, Parini, Rossella, Robinson, Amy, Schubert Hjalmarsson, Elke, White, Klane K., Wijburg, Frits A., Kuiper, Gé Ann, Langereis, Eveline J., Breyer, Sandra, Carbone, Marco, Castelein, René M., Eastwood, Deborah M., Garin, Christophe, Guffon, Nathalie, Van Hasselt, Peter M., Hensman, Pauline, Jones, Simon A., Kenis, Vladimir, Kruyt, Moyo, Van Der Lee, Johanna H., Mackenzie, William G., Orchard, Paul J., Oxborrow, Neil, Parini, Rossella, Robinson, Amy, Schubert Hjalmarsson, Elke, White, Klane K., and Wijburg, Frits A.

Published
2019

8. Synapse alterations precede neuronal damage and storage pathology in a human cerebral organoid model of CLN3-juvenile neuronal ceroid lipofuscinosis

Author

Metabole ziekten onderzoek 1, Cluster C, Metabole ziekten patientenzorg, Child Health, Gomez-Giro, Gemma, Arias-Fuenzalida, Jonathan, Jarazo, Javier, Zeuschner, Dagmar, Ali, Muhammad, Possemis, Nina, Bolognin, Silvia, Halder, Rashi, Jäger, Christian, Kuper, Willemijn F.E., Van Hasselt, Peter M., Zaehres, Holm, Del Sol, Antonio, Van Der Putten, Herman, Schöler, Hans R., Schwamborn, Jens C., Metabole ziekten onderzoek 1, Cluster C, Metabole ziekten patientenzorg, Child Health, Gomez-Giro, Gemma, Arias-Fuenzalida, Jonathan, Jarazo, Javier, Zeuschner, Dagmar, Ali, Muhammad, Possemis, Nina, Bolognin, Silvia, Halder, Rashi, Jäger, Christian, Kuper, Willemijn F.E., Van Hasselt, Peter M., Zaehres, Holm, Del Sol, Antonio, Van Der Putten, Herman, Schöler, Hans R., and Schwamborn, Jens C.

Published
2019

9. Emotional and behavioral problems, quality of life and metabolic control in NTBC-treated Tyrosinemia type 1 patients

Author

Cluster C, Metabole ziekten patientenzorg, Child Health, van Vliet, Kimber, van Ginkel, Willem G., Jahja, Rianne, Daly, Anne, MacDonald, Anita, De Laet, Corinne, Vara, Roshni, Rahman, Yusof, Cassiman, David, Eyskens, Francois, Timmer, Corrie, Mumford, Nicky, Bierau, Jörgen, van Hasselt, Peter M., Gissen, Paul, Goyens, Philippe J., McKiernan, Patrick J., Wilcox, Gisela, Morris, Andrew A.M., Jameson, Elisabeth A., Huijbregts, Stephan C.J., van Spronsen, Francjan J., Cluster C, Metabole ziekten patientenzorg, Child Health, van Vliet, Kimber, van Ginkel, Willem G., Jahja, Rianne, Daly, Anne, MacDonald, Anita, De Laet, Corinne, Vara, Roshni, Rahman, Yusof, Cassiman, David, Eyskens, Francois, Timmer, Corrie, Mumford, Nicky, Bierau, Jörgen, van Hasselt, Peter M., Gissen, Paul, Goyens, Philippe J., McKiernan, Patrick J., Wilcox, Gisela, Morris, Andrew A.M., Jameson, Elisabeth A., Huijbregts, Stephan C.J., and van Spronsen, Francjan J.

Published
2019

10. Treatment of thoracolumbar kyphosis in patients with mucopolysaccharidosis type I: Results of an international consensus procedure

Author

UMC Utrecht, MS Orthopaedie Algemeen, Regenerative Medicine and Stem Cells, Metabole ziekten patientenzorg, Child Health, Cluster C, Kuiper, Gé Ann, Langereis, Eveline J., Breyer, Sandra, Carbone, Marco, Castelein, René M., Eastwood, Deborah M., Garin, Christophe, Guffon, Nathalie, Van Hasselt, Peter M., Hensman, Pauline, Jones, Simon A., Kenis, Vladimir, Kruyt, Moyo, Van Der Lee, Johanna H., Mackenzie, William G., Orchard, Paul J., Oxborrow, Neil, Parini, Rossella, Robinson, Amy, Schubert Hjalmarsson, Elke, White, Klane K., Wijburg, Frits A., UMC Utrecht, MS Orthopaedie Algemeen, Regenerative Medicine and Stem Cells, Metabole ziekten patientenzorg, Child Health, Cluster C, Kuiper, Gé Ann, Langereis, Eveline J., Breyer, Sandra, Carbone, Marco, Castelein, René M., Eastwood, Deborah M., Garin, Christophe, Guffon, Nathalie, Van Hasselt, Peter M., Hensman, Pauline, Jones, Simon A., Kenis, Vladimir, Kruyt, Moyo, Van Der Lee, Johanna H., Mackenzie, William G., Orchard, Paul J., Oxborrow, Neil, Parini, Rossella, Robinson, Amy, Schubert Hjalmarsson, Elke, White, Klane K., and Wijburg, Frits A.

Published
2019

11. Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure.

Author

Langereis, Eveline J., Borgo, Andrea, Crushell, Ellen, Harmatz, Paul R., van Hasselt, Peter M., Jones, Simon A., Kelly, Paula M., Lampe, Christina, van der Lee, Johanna H., Odent, Thierry, Sakkers, Ralph, Scarpa, Maurizio, Schafroth, Matthias U., Struijs, Peter A., Valayannopoulos, Vassili, White, Klane K., and Wijburg, Frits A.

Abstract

Background: Mucopolysaccharidosis type I (MPS-I) is a lysosomal storage disorder characterized by progressive multi-organ disease. The standard of care for patients with the severe phenotype (Hurler syndrome, MPS I-H) is early hematopoietic stem cell transplantation (HSCT). However, skeletal disease, including hip dysplasia, is almost invariably present in MPS I-H, and appears to be particularly unresponsive to HSCT. Hip dysplasia may lead to pain and loss of ambulation, at least in a subset of patients, if left untreated. However, there is a lack of evidence to guide the development of clinical guidelines for the follow-up and treatment of hip dysplasia in patients with MPS I-H. Therefore, an international Delphi consensus procedure was initiated to construct consensus-based clinical practice guidelines in the absence of available evidence. Methods: A literature review was conducted, and publications were graded according to their level of evidence. For the development of consensus guidelines, eight metabolic pediatricians and nine orthopedic surgeons with experience in the care of MPS I patients were invited to participate. Eleven case histories were assessed in two written rounds. For each case, the experts were asked if they would perform surgery, and they were asked to provide information on the aspects deemed essential or complicating in the decision-making process. In a subsequent face-to-face meeting, the results were presented and discussed. Draft consensus statements were discussed and adjusted until consensus was reached. Results: Consensus was reached on seven statements. The panel concluded that early corrective surgery for MPS I-H patients with hip dysplasia should be considered. However, there was no full consensus as to whether such a procedure should be offered to all patients with hip dysplasia to prevent complications or whether a more conservative approach with surgical intervention only in those patients who develop clinically relevant symptoms due to the hip dysplasia is warranted. Conclusions: This international consensus procedure led to the construction of clinical practice guidelines for hip dysplasia in transplanted MPS I-H patients. Early corrective surgery should be considered, but further research is needed to establish its efficacy and role in the treatment of hip dysplasia as seen in MPS I. [ABSTRACT FROM AUTHOR]

Published
2013
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