Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience.
Department of Pediatric Neurology, Center for Childhood White Matter Disorders, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands;
Diane F
DF
van Rappard
Department of Pediatrics, Blood and Marrow Transplantation Program, Laboratory of Translational Immunology and Department of Hematology, and Sylvia Toth Center for Multidisciplinary Follow up after Hematopoietic Cell Transplantation, University Medical Center Utrecht, Utrecht, The Netherlands;
Jaap J
JJ
Boelens
Department of Pediatric Neurology, Center for Childhood White Matter Disorders, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands; University of Groningen, Department of Neurology, University Medical Center Groningen, Groningen, The Netherlands; Department of Neurology, Ommelander Ziekenhuis Groningen, Delfzijl and Winschoten, The Netherlands;
Martje E
ME
van Egmond
Laboratory of Translational Immunology and Department of Hematology, and.
Jurgen
J
Kuball
Sylvia Toth Center for Multidisciplinary Follow up after Hematopoietic Cell Transplantation, University Medical Center Utrecht, Utrecht, The Netherlands; Department of Pediatric Metabolic Disorders, University Medical Center Utrecht, Utrecht, The Netherlands;
Peter M
PM
van Hasselt
Department of Pediatric Psychology, VU University Medical Center Amsterdam, Amsterdam, The Netherlands;
Kim J
KJ
Oostrom
Department of Physics and Medical Technology, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands; and.
Petra J W
PJ
Pouwels
Department of Pediatric Neurology, Center for Childhood White Matter Disorders, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands;
Marjo S
MS
van der Knaap
Division of Endocrinology and Metabolism, Department of Internal Medicine, Academic Medical Center Amsterdam, Amsterdam, The Netherlands.
Carla E M
CE
Hollak
Department of Pediatric Neurology, Center for Childhood White Matter Disorders, VU University Medical Center, and Neuroscience Campus Amsterdam, Amsterdam, The Netherlands;
Nicole I
0000-0003-1721-0728
NI
Wolf
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D003071:Cognition; D015331:Cohort Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007966:Leukodystrophy, Metachromatic / Q000453:epidemiology / Q000503:physiopathology / Q000628:therapy*; D009426:Netherlands / Q000453:epidemiology; D016019:Survival Analysis; D016896:Treatment Outcome; D055815:Young Adult