• Medientyp: E-Artikel
  • Titel: Tandem duplication of DMD exon 18 associated with epilepsy, macroglossia, and endocrinologic abnormalities
  • Beteiligte: Weiss, Claudia; Jakubiczka, Sibylle; Huebner, Angela; Klopocki, Eva; Kress, Wolfram; Voit, Thomas; Hübner, Christoph; Schuelke, Markus
  • Erschienen: Wiley, 2007
  • Erschienen in: Muscle & Nerve
  • Sprache: Englisch
  • DOI: 10.1002/mus.20705
  • ISSN: 0148-639X; 1097-4598
  • Schlagwörter: Physiology (medical) ; Cellular and Molecular Neuroscience ; Neurology (clinical) ; Physiology
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  • Beschreibung: <jats:title>Abstract</jats:title><jats:p>We describe a patient with Duchenne muscular dystrophy (DMD) who additionally suffered from intractable seizures, severe mental retardation, and a marked macroglossia. He also had endocrinologic abnormalities consisting of growth hormone deficiency, delayed puberty, and adrenal hypoplasia. We detected a duplication of <jats:italic>DMD</jats:italic> exon 18 and flanking introns that caused a frame‐shift and was not removed by corrective splicing. A coincident mutation in the <jats:italic>FKRP</jats:italic> gene was excluded by direct sequencing. Complex DNA rearrangements, deletions, and duplications &gt;100 kb were excluded through microarray–comparative genomic hybridization (CGH), although we were not able to exclude a second coincident mutation with certainty. In conclusion, we present a case of DMD that conflicts with current understanding of genotype–phenotype relations and discuss putative pathogenetic mechanisms for this uncommon phenotype. Muscle Nerve, 2006</jats:p>