Allogenic hematopoietic stem cell transplantation in two siblings with adult metachromatic leukodystrophy and a systematic literature review

Abstract Two siblings were diagnosed with adult metachromatic leukodystrophy (MLD) and treated with hematopoietic stem cell transplantation (HSCT). While the older sibling was symptomatic at the time of diagnosis, her younger brother was diagnosed and transplanted at the presymptomatic state. We des...

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Bibliographic Details
Main Authors: Cecilie Videbæk, Jette Stokholm, Henrik Sengeløv, Lone U. Fjeldborg, Vibeke Andrée Larsen, Christian Krarup, Jørgen E. Nielsen, Sabine Grønborg
Format: Article
Language:English
Published: Wiley 2021-07-01
Series:JIMD Reports
Subjects:
Online Access:https://doi.org/10.1002/jmd2.12221