Zobrazeno 1 - 10
of 393
pro vyhledávání: '"Jaap-Jan Boelens"'
Autor:
Daphne H. Schoenmakers, Fanny Mochel, Laura A. Adang, Jaap-Jan Boelens, Valeria Calbi, Erik A. Eklund, Sabine W. Grønborg, Francesca Fumagalli, Samuel Groeschel, Caroline Lindemans, Caroline Sevin, Ludger Schöls, Dipak Ram, Ayelet Zerem, Holm Graessner, Nicole I. Wolf
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 19, Iss 1, Pp 1-9 (2024)
Abstract Background For decades, early allogeneic stem cell transplantation (HSCT) has been used to slow neurological decline in metachromatic leukodystrophy (MLD). There is lack of consensus regarding who may benefit, and guidelines are lacking. Cli
Externí odkaz:
https://doaj.org/article/15b67733f9df4ad2852654ef151aeadb
Autor:
Murtadha Al‐Saady, Shanice Beerepoot, Bonnie C. Plug, Marjolein Breur, Hristina Galabova, Petra J. W. Pouwels, Jaap‐Jan Boelens, Caroline Lindemans, Peter M. vanHasselt, Ulrich Matzner, Adeline Vanderver, Marianna Bugiani, Marjo S. van derKnaap, Nicole I. Wolf
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 10, Iss 7, Pp 1146-1159 (2023)
Abstract Objective Metachromatic leukodystrophy is a lysosomal storage disease caused by deficient arylsulfatase A. It is characterized by progressive demyelination and thus mainly affects the white matter. Hematopoietic stem cell transplantation may
Externí odkaz:
https://doaj.org/article/451b051a73834de09b3e42f76f2a7a8b
Autor:
Daphne H. Schoenmakers, Shanice Beerepoot, Sibren van den Berg, Laura Adang, Annette Bley, Jaap-Jan Boelens, Francesca Fumagalli, Wim G. Goettsch, Sabine Grønborg, Samuel Groeschel, Peter M. van Hasselt, Carla E. M. Hollak, Caroline Lindemans, Fanny Mochel, Peter G. M. Mol, Caroline Sevin, Ayelet Zerem, Ludger Schöls, Nicole I. Wolf
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 17, Iss 1, Pp 1-14 (2022)
Abstract Background Metachromatic Leukodystrophy (MLD) is a rare lysosomal disorder. Patients suffer from relentless neurological deterioration leading to premature death. Recently, new treatment modalities, including gene therapy and enzyme replacem
Externí odkaz:
https://doaj.org/article/8c393f5208e146328f0af2066de15352
Autor:
Celina L. Szanto, Annelisa M. Cornel, Sara M. Tamminga, Eveline M. Delemarre, Coco C. H. de Koning, Denise A. M. H. van den Beemt, Ester Dunnebach, Michelle L. Tas, Miranda P. Dierselhuis, Lieve G. A. M. Tytgat, Max M. van Noesel, Kathelijne C. J. M. Kraal, Jaap-Jan Boelens, Alwin D. R. Huitema, Stefan Nierkens
Publikováno v:
Cancers, Vol 13, Iss 9, p 2096 (2021)
Despite intensive treatment, including consolidation immunotherapy (IT), prognosis of high-risk neuroblastoma (HR-NBL) is poor. Immune status of patients over the course of treatment, and thus immunological features potentially explaining therapy eff
Externí odkaz:
https://doaj.org/article/28aaf9cbe94446c1b203e7960566c9cd
Autor:
Katarzyna B. Gostyńska, Vamsi K. Yenamandra, Caroline Lindemans, José Duipmans, Antoni Gostyński, Marcel F. Jonkman, Jaap-Jan Boelens
Publikováno v:
Acta Dermato-Venereologica, Vol 99, Iss 3, Pp 347-348 (2018)
Externí odkaz:
https://doaj.org/article/013c9f9d3ebc45c6b1c15485bac672c8
Autor:
Agata Drewniak, Jaap-Jan Boelens, Hans Vrielink, Anton T.J. Tool, Marrie C.A. Bruin, Marry van den Heuvel-Eibrink, Lynne Ball, Marianne D. van de Wetering, Dirk Roos, Taco W. Kuijpers
Publikováno v:
Haematologica, Vol 93, Iss 7 (2008)
Background Granulocyte transfusion has been proposed as a bridging therapy for patients with prolonged periods of chemotherapy-induced neutropenia, who suffer from severe fungal and bacterial infections. To recover, adequate numbers of granulocytes a
Externí odkaz:
https://doaj.org/article/36c7364550294f8cbc70fc9062b94b14
Autor:
Moises Garcia-Rosa, Allistair Abraham, Alice Bertaina, Senthil Velan Bhoopalan, Carmem Bonfim, Sandra Cohen, Amy DeZern, Chrystal Louis, Joseph Oved, Mara Pavel-Dinu, Duncan Purtill, Annalisa Ruggeri, Athena Russell, Akshay Sharma, Robert Wynn, Jaap Jan Boelens, Susan Prockop
Publikováno v:
Cytotherapy. 25:578-589
Autor:
Shanice Beerepoot, Stefan Nierkens, Jaap Jan Boelens, Caroline Lindemans, Marianna Bugiani, Nicole I. Wolf
Publikováno v:
Orphanet Journal of Rare Diseases, Vol 14, Iss 1, Pp 1-13 (2019)
Abstract Metachromatic leukodystrophy (MLD) is an autosomal recessively inherited metabolic disease characterized by deficient activity of the lysosomal enzyme arylsulfatase A. Its deficiency results in accumulation of sulfatides in neural and viscer
Externí odkaz:
https://doaj.org/article/7fd5539a372f4a278d7da2defbd52fd2
Autor:
Maud Plantinga, Vania Lo Presti, Colin G. de Haar, Ester Dünnebach, Alejandro Madrigal, Caroline A. Lindemans, Jaap Jan Boelens, Stefan Nierkens
Publikováno v:
Frontiers in Immunology, Vol 11 (2020)
Hematopoietic cell transplantation (HCT) is a last resort, potentially curative treatment option for pediatric patients with refractory acute myeloid leukemia (AML). Cord blood transplantation (CBT) results in less relapses and less graft-versus-host
Externí odkaz:
https://doaj.org/article/ae61d80aa4024b78b602ce6cc78c2473
Publikováno v:
Frontiers in Pediatrics, Vol 8 (2020)
Hematopoietic cell transplantation (HCT) is often a last resort, but potentially curative treatment option for children suffering from hematological malignancies and a variety of non-malignant disorders, such as bone marrow failure, inborn metabolic
Externí odkaz:
https://doaj.org/article/0f0d471dd4394bba828185db951e6400