Bone marrow transplantation in glycogen storage disease type 1b.

Pierre, Germaine; Chakupurakal, Geothy; McKiernan, Patrick; Hendriksz, Chris; Lawson, Sarah; Chakrapani, Anupam · J Pediatr · 2008

case_report · Level V

Where this comes from

Abstract

We report a case of glycogen storage disease type 1b that was successfully treated with bone marrow transplantation after life-threatening complications related to neutropenia and thrombocytopenia. Concomitant reduction in inflammatory bowel disease-related symptoms and improved metabolic stability were also observed.

Medical subject headings