Literature DB >> 11971051

Familial adult onset of Krabbe's disease resembling hereditary spastic paraplegia with normal neuroimaging.

N P S Bajaj1, A Waldman, R Orrell, N W Wood, K P Bhatia.   

Abstract

Krabbe's disease (globoid cell leucodystrophy) is a disorder involving the white matter of the peripheral and central nervous systems. Mutations in the gene for the lysosomal enzyme galactocerebrosidase (GALC) result in low enzymatic activity and decreased ability to degrade galactolipids found in myelin. The disease is classically of infantile onset, but adult onset cases have been reported. Magnetic resonance imaging (MRI) of the brain shows characteristic abnormalities. A unique family with Krabbe's disease is described, with proven GALC deficiency but normal MRI. A neurological phenotype is present in heterozygotes and the family shows the extent of homozygotic phenotypic diversity that can be seen in this disorder.

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Year:  2002        PMID: 11971051      PMCID: PMC1737880          DOI: 10.1136/jnnp.72.5.635

Source DB:  PubMed          Journal:  J Neurol Neurosurg Psychiatry        ISSN: 0022-3050            Impact factor:   10.154


  11 in total

1.  Peripheral neuropathy in late-onset Krabbe disease: report of three cases.

Authors:  A Malandrini; C D'Eramo; S Palmeri; C Gaudiano; S Gambelli; F Sicurelli; G Berti; P Formichi; A Kuqo; M T Dotti; A Federico
Journal:  Neurol Sci       Date:  2012-01-25       Impact factor: 3.307

2.  Evidence for improved survival in postsymptomatic stem cell-transplanted patients with Krabbe's disease.

Authors:  Thomas J Langan; Amy L Barcykowski; Jonathan Dare; Erin C Pannullo; Leah Muscarella; Randy L Carter
Journal:  J Neurosci Res       Date:  2016-11       Impact factor: 4.164

Review 3.  Hereditary spastic paraparesis in adults associated with inborn errors of metabolism: a diagnostic approach.

Authors:  F Sedel; B Fontaine; J M Saudubray; O Lyon-Caen
Journal:  J Inherit Metab Dis       Date:  2007-10-22       Impact factor: 4.982

4.  Two Cases of Female Chinese Adult-Onset Krabbe Disease with One Novel Mutation and a Review of Literature.

Authors:  Chengyi Zhang; Zheng Liu; Huiqing Dong
Journal:  J Mol Neurosci       Date:  2020-11-14       Impact factor: 3.444

Review 5.  Lysosomal storage diseases.

Authors:  Carlos R Ferreira; William A Gahl
Journal:  Transl Sci Rare Dis       Date:  2017-05-25

6.  Krabbe disease in adults: phenotypic and genotypic update from a series of 11 cases and a review.

Authors:  Rabab Debs; Roseline Froissart; Patrick Aubourg; Caroline Papeix; Claire Douillard; Bertrand Degos; Bertrand Fontaine; Bertrand Audoin; Arnaud Lacour; Gérard Said; Marie T Vanier; Frédéric Sedel
Journal:  J Inherit Metab Dis       Date:  2012-11-30       Impact factor: 4.982

Review 7.  Lipid Involvement in Neurodegenerative Diseases of the Motor System: Insights from Lysosomal Storage Diseases.

Authors:  James C Dodge
Journal:  Front Mol Neurosci       Date:  2017-11-03       Impact factor: 5.639

8.  Late-Onset Leukodystrophy Mimicking Hereditary Spastic Paraplegia without Diffuse Leukodystrophy on Neuroimaging.

Authors:  Tongxia Zhang; Chuanzhu Yan; Yiming Liu; Lili Cao; Kunqian Ji; Duoling Li; Lingyi Chi; Yuying Zhao
Journal:  Neuropsychiatr Dis Treat       Date:  2021-05-12       Impact factor: 2.570

Review 9.  Leukoencephalopathies associated with inborn errors of metabolism in adults.

Authors:  F Sedel; A Tourbah; B Fontaine; C Lubetzki; N Baumann; J-M Saudubray; O Lyon-Caen
Journal:  J Inherit Metab Dis       Date:  2008-02-25       Impact factor: 4.750

10.  Adult-onset Krabbe disease due to a homozygous GALC mutation without abnormal signals on an MRI in a consanguineous family: A case report.

Authors:  Zhou Xia; Yin Wenwen; Yu Xianfeng; Hu Panpan; Zhu Xiaoqun; Sun Zhongwu
Journal:  Mol Genet Genomic Med       Date:  2020-07-17       Impact factor: 2.183

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