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Citation(s)

  •   Cantor RM, Roy C, Lim JS, Kaback MM
    Sandhoff disease heterozygote detection: a component of population screening for Tay-Sachs disease carriers. II. Sandhoff disease gene frequencies in American Jewish and non-Jewish populations. Am J Hum Genet. 1987 Jul;41(1):16-26.
  •   Myerowitz R, Hogikyan ND
    Different mutations in Ashkenazi Jewish and non-Jewish French Canadians with Tay-Sachs disease. Science. 1986 Jun 27;232(4758):1646-8. doi: 10.1126/science.3754980.
  •   Wada R, Tifft CJ, Proia RL
    Microglial activation precedes acute neurodegeneration in Sandhoff disease and is suppressed by bone marrow transplantation. Proc Natl Acad Sci U S A. 2000 Sep 26;97(20):10954-9. doi: 10.1073/pnas.97.20.10954.

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Details for clinical trial NCT00029965