Dysarthria and Stutter as Presenting Symptoms of Late-Onset Tay-Sachs Disease in Three Siblings

被引:9
作者
Grim, Kristina K. [1 ]
Phillips, Gregory D. [1 ]
Renner, David R. [1 ]
机构
[1] Univ Utah, Dept Neurol, 175 N Med Dr,5th Floor Easr, Salt Lake City, UT 84132 USA
关键词
Tay-Sachs disease; hexosaminidase A; G(M2) gangliosidosis; stutter; dysarthria;
D O I
10.1002/mdc3.12194
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
Late-onset Tay-Sachs disease (LOTS) is a rare autosomal-recessive genetic disorder caused by insufficient activity of the lysosomal enzyme, beta-hexosaminidase A, resulting in intracellular accumulation of gangliosides in the central nervous system. Clinical manifestations can include unsteadiness in gait, muscle weakness, cognitive dysfunction, psychiatric disturbance, and dysarthric speech. The variable presentation of these symptoms, combined with the late onset of the disease, often results in misdiagnosis. We present video of 3 sibling cases of LOTS in which a dysarthric stutter was the sole presenting symptom in order to better characterize the phenotype of this disease.
引用
收藏
页码:289 / 290
页数:2
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