Identification of Sandhoff disease in a Thai family: clinical and biochemical characterization.

نویسندگان

  • Kullasate Sakpichaisakul
  • Pairat Taeranawich
  • Achara Nitiapinyasakul
  • Todsaporn Sirisopikun
چکیده

Sandhoff disease is a GM2 gangliosidosis that is rare in Thailand. The authors report a Thai family with two children known to have infantile form of Sandhoff disease. The index case exhibited mitral valve prolapse with mitral regurgitation as an early sign, which is a rare presentation in Sandhoff disease. Thereafter the patient had developmental regression, startle reaction, and cherry red spots. The diagnosis was confirmed by biochemical analysis.

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عنوان ژورنال:
  • Journal of the Medical Association of Thailand = Chotmaihet thangphaet

دوره 93 9  شماره 

صفحات  -

تاریخ انتشار 2010