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Juvenile metachromatic leukodystrophy: neurological outcome two years after bone marrow transplantation
Author(s) -
Guffon N.,
Souillet G.,
Maire I.,
Dorche C.,
Mathieu M.,
Guibaud P.
Publication year - 1995
Publication title -
journal of inherited metabolic disease
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.462
H-Index - 102
eISSN - 1573-2665
pISSN - 0141-8955
DOI - 10.1007/bf00711755
Subject(s) - metachromatic leukodystrophy , medicine , sibling , asymptomatic , hurler syndrome , leukodystrophy , transplantation , bone marrow transplantation , surgery , pediatrics , pathology , psychology , disease , developmental psychology
Conclusion Our patient was not an ideal candidate since she was not clinically completely asymptomatic and presented with abnormal MRI and decreased nerve conduction when the metachromatic leukodystrophy was diagnosed in her older sibling. Nevertheless, 2 years after graft the deterioration did not progress and the MRI improved, while her brother became bed‐ridden within few months after the same initial symptoms. These favourable results need further confirmation after several years since significant differences in course have been observed in siblings (Clarke et al 1989).