Early-onset ALS with long-term survival associated with spastin gene mutation.

Meyer, T; Schwan, A; Dullinger, J S; Brocke, J; Hoffmann, K-T; Nolte, C H; Hopt, A; Kopp, U et al. · Neurology · 2005

case_report · Level V

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Abstract

The authors report a 73-year-old patient with a natural history of early-onset ALS for 49 years presenting with limb and bulbar amyotrophy and a pyramidal syndrome. Analysis of the locus SPG4 identified a heterozygous duplication mutation (c.304_309dupGCCTCG) within exon 1 of the spastin gene. We propose that sequence alterations of spastin may comprise a genetic risk factor in a greater spectrum of motor neuron disorders including clinical variants of ALS.

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