Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA☆
Mucopolysaccharidosis IVA (MPS IVA) is one of the lysosomal storage diseases. It is caused by the deficiency of N-acetylgalactosamine-6-sulfate sulfatase. Deficiency of this enzyme leads to accumulation of the specific glycosaminoglycans keratan sulfate and chondroitin-6-sulfate. This accumulation h...
Main Authors: | , , , , , |
---|---|
Format: | Online |
Language: | English |
Published: |
Elsevier
2014
|
Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4292891/ |