Allogeneic haematopoietic stem cell transplantation in late-onset metachromatic leukodystrophy.

D. Schoenmakers M. Datema Shanice Beerepoot Marije A. B. C. Asbreuk C. Bergner C. Awißus Annette Bley H. Sigel Janna Bredow V. Calbi F. Fumagalli Emilia Inverso Erik A. Eklund S. Grønborg W. Grol Cecilie S. Videbæk C. Kehrer M. S. van der Knaap Nick Schnermann H. Rosewich Peter Lang P. V. van Hasselt Caroline Lindemans M. D. de Witte M. Langeveld L. Schöls J. Boelens Päivi M. Vieira J. Uusimaa Fanny Mochel Caroline Sevin C. Hollak S. Groeschel N. Wolf

Sep 2026 · Brain : a journal of neurology · 0 citations

Medicine

TL;DR

Comparing the long-term outcomes of the largest European cohort of HSCT-treated and untreated late-juvenile and adult MLD patients provides an evidence base for future comparison with innovative therapies and underscores the importance of early diagnosis and intervention.

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