Galactosemia Pediatric Research and Clinical Trials

LIke ThisLIke ThisLIke ThisLIke ThisLIke This

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.

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

Boston Children's Hospital
300 Longwood Avenue, Boston, MA 02115
For Patients: 617-355-6000
For Referring Providers: 844-BCH-PEDS | 844-224-7337