Biochemical and clinical response after umbilical cord blood transplant in a boy with early childhood ‐onset beta‐mannosidosis

ConclusionOur findings suggest that hematopoietic cell transplant can correct the biochemical defect in β‐mannosidosis, although preservation of the neurocognitive trajectory may be a challenge.

Source: Molecular Genetics & Genomic Medicine - May 20, 2019 Category: Genetics & Stem Cells Authors: Troy C. Lund, Weston P. Miller, Julie B. Eisengart, Katrina Simmons, Laura Pollard, Deborah L. Renaud, David A. Wenger, Marc C. Patterson, Paul J Orchard Tags: ORIGINAL ARTICLE Source Type: research