In rare, hereditary storage diseases such as Sandhoff’s disease or Tay-Sachs syndrome, the metabolic waste from accumulating gangliosides cannot be properly disposed of in the nerve cells because important enzymes are missing. The consequences are grave: They range from movement restrictions to blindness, mental decline and early death. Scientists now demonstrate why these gangliosides also accumulate in patients with other storage diseases and cause a deterioration in them.
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