Allogeneic Hematopoietic Cell Transplantation for Morquio A Syndrome: An International Retrospective Study
Sandhya Kharbanda, Emma Cho, Jing Chen, Manpin Zhang et autres
BackgroundMucopolysaccharidosis IV A (MPS IVA) or Morquio A syndrome is a lysosomal storage disorder that results from a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS), causing deposition of glycosaminoglycans, keratan sulfate, and chondroitin-6-sulfate in multiple organs. The main clinical manifestations include severe short stature, …
0 citations
Utrecht University Repository (Utrecht University)