Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective
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Title
Peripheral neuropathy in metachromatic leukodystrophy: current status and future perspective
Authors
Keywords
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Journal
Orphanet Journal of Rare Diseases
Volume 14, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2019-11-04
DOI
10.1186/s13023-019-1220-4
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Note: Only part of the references are listed.- Evolutionary redesign of the lysosomal enzyme arylsulfatase A increases efficacy of enzyme replacement therapy for metachromatic leukodystrophy
- (2019) Heidi Simonis et al. HUMAN MOLECULAR GENETICS
- Intrathecal baclofen in metachromatic leukodystrophy
- (2018) Nikki Van Der Veldt et al. DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY
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- (2018) Allison M. Bradbury et al. HUMAN GENE THERAPY
- Intracerebral gene therapy in children with metachromatic leukodystrophy: Results of a phase I/II trial
- (2018) Caroline Sevin et al. MOLECULAR GENETICS AND METABOLISM
- Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I
- (2017) Rachele Penati et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Subacute demyelinating peripheral neuropathy as a novel presentation of late infantile metachromatic leukodystrophy
- (2017) Hernan D. Gonorazky et al. MUSCLE & NERVE
- Efficacy of hematopoietic cell transplantation in metachromatic leukodystrophy: the Dutch experience
- (2016) D. F. van Rappard et al. BLOOD
- The Structure-Function Relationships of Complement Receptor Type 2 (CR2; CD21)
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- Late-Onset Metachromatic Leukodystrophy with Early Onset Dementia Associated with a Novel Missense Mutation in the Arylsulfatase A Gene
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- Thickening of the peripheral nerves in metachromatic leukodystrophy
- (2016) Alexander Grimm et al. JOURNAL OF THE NEUROLOGICAL SCIENCES
- Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial
- (2016) Maria Sessa et al. LANCET
- Cerebral Spinal Fluid levels of Cytokines are elevated in Patients with Metachromatic Leukodystrophy
- (2016) Kathryn A. Thibert et al. Scientific Reports
- Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Juvenile Metachromatic Leukodystrophy Compared With Nontransplanted Control Patients
- (2016) Samuel Groeschel et al. JAMA Neurology
- Metachromatic leukodystrophy: Disease spectrum and approaches for treatment
- (2015) Diane F. van Rappard et al. BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM
- Sulfatide Analysis by Mass Spectrometry for Screening of Metachromatic Leukodystrophy in Dried Blood and Urine Samples
- (2015) Z. Spacil et al. CLINICAL CHEMISTRY
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- Mutation Update ofARSAandPSAPGenes Causing Metachromatic Leukodystrophy
- (2015) Martina Cesani et al. HUMAN MUTATION
- Outcome of Early Juvenile Onset Metachromatic Leukodystrophy After Unrelated Cord Blood Transplantation
- (2015) Xuqin Chen et al. JOURNAL OF CHILD NEUROLOGY
- Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features
- (2015) Mohammad A. Rafi et al. MOLECULAR GENETICS AND METABOLISM
- Anti-inflammatory Therapy With Simvastatin Improves Neuroinflammation and CNS Function in a Mouse Model of Metachromatic Leukodystrophy
- (2015) Axel Stein et al. MOLECULAR THERAPY
- Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report
- (2015) Alexander A. Boucher et al. Orphanet Journal of Rare Diseases
- Scavenger Receptor-A (CD204): A Two-Edged Sword in Health and Disease
- (2014) Jim L. Kelley et al. CRITICAL REVIEWS IN IMMUNOLOGY
- Etiologic Spectrum of Biopsy-Proven Peripheral Neuropathies in Childhood from a Resource-Poor Setting
- (2014) Pramod Krishnan et al. JOURNAL OF CHILD NEUROLOGY
- 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
- Leukodystrophy Presenting as Acute-Onset Polyradiculoneuropathy
- (2014) Rachana Dubey et al. PEDIATRIC NEUROLOGY
- Molecular triggers of neuroinflammation in mouse models of demyelinating diseases
- (2013) Benoit Barrette et al. BIOLOGICAL CHEMISTRY
- Neurodevelopmental Outcomes of Umbilical Cord Blood Transplantation in Metachromatic Leukodystrophy
- (2013) Holly R. Martin et al. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION
- Advances and Pitfalls of Cell Therapy in Metabolic Leukodystrophies
- (2013) Catarina Oliveira Miranda et al. CELL TRANSPLANTATION
- Interleukin-8 Regulates Endothelial Permeability by Down-regulation of Tight Junction but not Dependent on Integrins Induced Focal Adhesions
- (2013) Hongchi Yu et al. International Journal of Biological Sciences
- Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
- (2013) A. Biffi et al. SCIENCE
- Juvenile metachromatic leukodystrophy 10 years post transplant compared with a non-transplanted cohort
- (2012) I Krägeloh-Mann et al. BONE MARROW TRANSPLANTATION
- Correction of Brain Oligodendrocytes by AAVrh.10 Intracerebral Gene Therapy in Metachromatic Leukodystrophy Mice
- (2012) Françoise Piguet et al. HUMAN GENE THERAPY
- Efficacy of enzyme replacement therapy in an aggravated mouse model of metachromatic leukodystrophy declines with age
- (2012) F. Matthes et al. HUMAN MOLECULAR GENETICS
- Biology of the blood–nerve barrier and its alteration in immune mediated neuropathies
- (2012) Takashi Kanda JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
- The pediatric-modified total neuropathy score: a reliable and valid measure of chemotherapy-induced peripheral neuropathy in children with non-CNS cancers
- (2012) Laura S. Gilchrist et al. SUPPORTIVE CARE IN CANCER
- Adult metachromatic leukodystrophy treated by allo-SCT and a review of the literature
- (2010) L D de Hosson et al. BONE MARROW TRANSPLANTATION
- Demyelination and axonal preservation in a transgenic mouse model of Pelizaeus-Merzbacher disease
- (2010) Julia M. Edgar et al. EMBO Molecular Medicine
- Unrelated umbilical cord blood transplant for juvenile metachromatic leukodystrophy: A 5-year follow-up in three affected siblings
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- Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation
- (2009) A Biffi et al. CLINICAL GENETICS
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- (2009) Rani K. Singh et al. PEDIATRIC NEUROLOGY
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- Expanded Repertoire of AAV Vector Serotypes Mediate Unique Patterns of Transduction in Mouse Brain
- (2008) Cassia N Cearley et al. MOLECULAR THERAPY
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