Zobrazeno 1 - 10
of 20
pro vyhledávání: '"Caroline, Lindemans"'
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:
Anna L David, Mats Johansson, Magnus Westgren, Belinda Crowe, Dick Oepkes, Melissa Hill, Lyn S Chitty, Catherine DeVile, Peter Lindgren, Eva Åström, Cecilia Götherström, Nils-Eric Sahlin, Rachel L Sagar, Annabelle Forsmark, Vera Franzen, Göran Hermeren, Caroline Lindemans, Wouter Nijhuis, Mirko Rehberg, Ralph Sakkers, O Semler, Mikael Sundin, Lilian Walther-Jallow, E J T Joanne Verweij
Publikováno v:
BMJ Open, Vol 14, Iss 6 (2024)
Introduction Severe osteogenesis imperfecta (OI) is a debilitating disease with no cure or sufficiently effective treatment. Mesenchymal stem cells (MSCs) have good safety profile, show promising effects and can form bone. The Boost Brittle Bones Bef
Externí odkaz:
https://doaj.org/article/582a3f441343419db9a89157809ffc1c
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:
Paul Harmatz, Robert Wynn, Ashish Gupta, Sandhya Kharbanda, Caroline Lindemans, Rebecca Ahrens-Nicklas, Peter van Hasselt, Troy Lund, Timothy Olson, Francesca Tucci, Leonie Martin, Nathalie Boeglin, Jean Brooks, Su Syonmez, Laura Campbell, Simon Jones, Paul Orchard, Maria Ester Bernardo
Publikováno v:
Genetics in Medicine Open, Vol 2, Iss , Pp 100914- (2024)
Externí odkaz:
https://doaj.org/article/70e16dbba1ce45d1b98e1211c66c59e6
Autor:
Eva Koopman-Coenen, Joyce Meesters-Ensing, Stefan Nierkens, Ireen Kal, Konradin Müskens, Birgitta Versluys, Caroline Lindemans, Marc Bierings, Rick Admiraal, Mirjam Belderbos
Publikováno v:
EJC Paediatric Oncology, Vol 2, Iss , Pp 100033- (2023)
Externí odkaz:
https://doaj.org/article/8b2759f31c4642d7b8faf8dd648fa12d
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:
Agnieszka Sobkowiak-Sobierajska, Caroline Lindemans, Tomas Sykora, Jacek Wachowiak, Jean-Hugues Dalle, Halvard Bonig, Andrew Gennery, Anita Lawitschka
Publikováno v:
Frontiers in Pediatrics, Vol 10 (2022)
Herein we review current practice regarding the management of chronic graft-vs.-host disease (cGvHD) in paediatric patients after allogeneic haematopoietic stem cell transplantation (HSCT) for acute lymphoblastic leukaemia (ALL). Topics covered inclu
Externí odkaz:
https://doaj.org/article/52b733eea58f4ac2b123b9c8ecbf6543
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:
Su Han Lum, Milen Minkov, Simon A. Jones, Sheree Hazelaar, Tiarlan Sirait, Jane E. Potter, Polina Stepensky, Frederic Garban, Herbert Pichler, Jerry Stein, Zuhre Kaya, Ansgar Schulz, Karin Mellgren, Cristina Diaz de Heredia, Cecile Pochon, Susana Riesco, Miguel Angel Diaz, Gérard Michel, Caroline Lindemans, Bernd Gruhn, Michael H. Albert, Arjan C. Lankester, Bénédicte Neven, Robert Wynn
Publikováno v:
Bone Marrow Transplantation. 58:594-596
Autor:
Alastair Baker, Esteban Frauca Remacha, Juan Torres Canizales, Luz Yadira Bravo-Gallego, Emer Fitzpatrick, Angel Alonso Melgar, Gema Muñoz Bartolo, Luis Garcia Guereta, Esther Ramos Boluda, Yasmina Mozo, Dorota Broniszczak, Wioletta Jarmużek, Piotr Kalicinski, Britta Maecker-Kolhoff, Julia Carlens, Ulrich Baumann, Charlotte Roy, Christophe Chardot, Elisa Benetti, Mara Cananzi, Elisabetta Calore, Luca Dello Strologo, Manila Candusso, Maria Francelina Lopes, Manuel João Brito, Cristina Gonçalves, Carmen Do Carmo, Xavier Stephenne, Lars Wennberg, Rosário Stone, Jelena Rascon, Caroline Lindemans, Dominik Turkiewicz, Eugenia Giraldi, Emanuele Nicastro, Lorenzo D’Antiga, Oanez Ackermann, Paloma Jara Vega
Publikováno v:
Children, Vol 8, Iss 8, p 661 (2021)
(1) Background: Post-transplant lymphoproliferative disease (PTLD) is a significant complication of solid organ transplantation (SOT). However, there is lack of consensus in PTLD management. Our aim was to establish a present benchmark for comparison
Externí odkaz:
https://doaj.org/article/e4cb0f49cb584054a82941ab9f84d7ba