Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report

Hematopoietic SCT: a useful treatment for late metachromatic leukodystrophy

(2014) M Solders et al.   BONE MARROW TRANSPLANTATION

Sulfatide, A Major Lipid Component of Myelin Sheath, Activates Inflammatory Responses As an Endogenous Stimulator in Brain-Resident Immune Cells

(2014) S.-B. Jeon et al.   JOURNAL OF IMMUNOLOGY

Language and cognition in children with metachromatic leukodystrophy: onset and natural course in a nationwide cohort

(2014) Christiane Kehrer et al.   Orphanet Journal of Rare Diseases

Neurodevelopmental Outcomes of Umbilical Cord Blood Transplantation in Metachromatic Leukodystrophy

(2013) Holly R. Martin et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Quantification of sulfatides in dried blood and urine spots from metachromatic leukodystrophy patients by liquid chromatography/electrospray tandem mass spectrometry

(2013) Mariana Barcenas et al.   CLINICA CHIMICA ACTA

Busulfan Conditioning Enhances Engraftment of Hematopoietic Donor-derived Cells in the Brain Compared With Irradiation

(2013) Fiona L Wilkinson et al.   MOLECULAR THERAPY

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Improvement of White Matter Changes on Neuroimaging Modalities After Stem Cell Transplant in Metachromatic Leukodystrophy

(2013) Martje E. van Egmond et al.   JAMA Neurology

Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort

(2012) I Krägeloh-Mann et al.   BONE MARROW TRANSPLANTATION

Cerebral gray and white matter changes and clinical course in metachromatic leukodystrophy

(2012) S. Groeschel et al.   NEUROLOGY

Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

(2012) A. Capotondo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The natural course of gross motor deterioration in metachromatic leukodystrophy

(2011) CHRISTIANE KEHRER et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Developing treatment options for metachromatic leukodystrophy

(2011) Spyros P. Batzios et al.   MOLECULAR GENETICS AND METABOLISM

Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature

(2010) L D de Hosson et al.   BONE MARROW TRANSPLANTATION

Development and reliability of a classification system for gross motor function in children with metachromatic leucodystrophy

(2010) CHRISTIANE KEHRER et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: A 5-year follow-up in three affected siblings

(2010) Casey Cable et al.   MOLECULAR GENETICS AND METABOLISM

Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation

(2009) A Biffi et al.   CLINICAL GENETICS

Metachromatic Leukodystrophy: A Case of Triplets With the Late Infantile Variant and a Systematic Review of the Literature

(2009) Asif Mahmood et al.   JOURNAL OF CHILD NEUROLOGY

Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options

(2008) Volkmar Gieselmann   ACTA PAEDIATRICA