Galactosemia Pediatric Research and Clinical Trials

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The Metabolism Program at Boston Children’s Hospital has one of the world’s largest and most advanced research programs in galactosemia.

One of the new, important activities in galactosemia research is to begin to create induced stem cells from the skin cells of patients with hereditary galactosemia. As these stem cells can be differentiated into brain cells, it will enable us to better study disease mechanisms. 

Researchers also hope to perform gene therapy on the differentiated brain cells as   a prelude to future studies that may include cell transplantation.

Learn more about research at Children's.

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