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The Molecular Pathogenesis of Pelizaeus-Merzbacher Disease
Journal article   Open access   Peer reviewed

The Molecular Pathogenesis of Pelizaeus-Merzbacher Disease

James Garbern, Franca Cambi, Michael Shy and John Kamholz
Archives of neurology (Chicago), Vol.56(10), pp.1210-1214
10/01/1999
DOI: 10.1001/archneur.56.10.1210
PMID: 10520936
url
https://doi.org/10.1001/archneur.56.10.1210View
Published (Version of record) Open Access

Abstract

In 1885, Pelizaeus described 5 boys in a single family with nystagmus, spastic quadriparesis, ataxia, and delay in cognitive development. In 1910, Merzbacher reexamined this family, which then included 14 affected individuals, including 2 girls, and found that all affected family members shared a common female ancestor. Also, he noted that the disease was passed exclusively through the female line without male-to-male transmission. Pathological analysis of brain tissue from one affected individual showed that most of the central white matter lacked histochemical staining for myelin, although there were occasional small regions of preserved myelin, giving the sections a "tigroid" appearance. The description of this family provides the clinical, genetic, and pathological basis for Pelizaeus-Merzbacher disease (PMD): an X-linked disorder of myelination classically characterized by nystagmus, spastic quadriparesis, ataxia, and cognitive delay in early childhood.

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