Laboratory modified corn has shown some progress in treating a rare, life-threatening childhood genetic disease. Researchers have been developing enzyme therapeutics for lysosomal storage diseases – rare but devastating childhood genetic diseases – for more than a decade. 

In the most severe forms of these inherited diseases, untreated patients die in early childhood because of progressive damage to all organs of the body and currently, enzyme treatments are available for only six of the more than 70 diverse types of lysosomal storage diseases.


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