Demyelination load as predictor for disease progression in juvenile metachromatic leukodystrophy

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Demyelination load as predictor for disease progression in juvenile metachromatic leukodystrophy

OBJECTIVE The aim of this study was to investigate whether the extent and topography of cerebral demyelination correlates with and predicts disease progression in patients with juvenile metachromatic leukodystrophy (MLD). METHODS A total of 137 MRIs of 46 patients with juvenile MLD were analyzed. Demyelination load and brain volume were quantified using the previously developed Software "clus...

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Juvenile metachromatic leukodystrophy in a boy with epilepsy.

M leukodystrophy (MLD) is an autosomal recessive disorder, caused by deficiency of arylsulfatase A (cerebroside sulfatase A, ASA), which results in accumulation of sulfatides, mainly in the brain and peripheral nerves. The estimated frequency of MLD from previous reports is 1 in 40,000 cases. The deficiency in ASA activity is caused by mutations in the ASA gene. The deficiency is systemic and a...

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Volumetric MRI data correlate to disease severity in metachromatic leukodystrophy

OBJECTIVE Metachromatic leukodystrophy (MLD) is an inherited lysosomal disorder due to a deficiency in arylsulfatase A with progressive demyelination and neurological decline. This retrospective MRI study investigated the extent of cortical involvement at time of diagnosis, and clinical correlates to both conventional and regional volumetric measures of brain involvement. METHODS 3D-T1-weight...

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MR of childhood metachromatic leukodystrophy.

PURPOSE To investigate the MR findings of childhood metachromatic leukodystrophy (MLD). METHODS Nine MR imaging studies in seven children (five girls and two boys, 10 to 32 months old) with MLD were evaluated retrospectively for the extent and progression of white matter abnormalities and the presence of contrast enhancement. RESULTS All seven cases showed symmetric, confluent high signal i...

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PSAP (prosaposin (variant Gaucher disease and variant metachromatic leukodystrophy))

The human PSAP-precursor gene spans approximately 20 kb in length of the long arm of chromosome 10 and consists at least 15 exons. The size of exons range from 57 to 1200 bp and the size of the introns vary from 91 to more than 3800 bp in length. The PSAP gene can be cateogorized as a polycistronic gene. Further analysis of PSAP intronic positions has indicated that it may be evolved from an an...

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ژورنال

عنوان ژورنال: Annals of Clinical and Translational Neurology

سال: 2017

ISSN: 2328-9503

DOI: 10.1002/acn3.420