X-linked myotubular myopathy in a female infant caused by a new MTM1 gene mutation.

Schara, Ulrike; Kress, Wolfram; Tücke, Jens; Mortier, Wilhelm · Neurology · 2003

case_report · Level V

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Abstract

X-linked myotubular myopathy usually affects male infants with a severe phenotype leading to early death or survival with severe handicaps. Female carriers have been reported manifesting in childhood with slowly progressive muscle weakness only. The authors describe a now 5-year-old girl with prenatal/neonatal onset of an X-linked myotubular myopathy due to a 605delT mutation in the myotubularin gene.

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