Emma Cho

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, …

Allogeneic Hematopoietic Cell Transplantation for Morquio A Syndrome: An International Retrospective Study

Sandhya Kharbanda, Emma Cho, Jing Chen, Manpin Zhang et autres

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Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Mucopolysaccharidosis IV a (Morquio A): An International Retrospective Study of 40 Children

Sandhya Kharbanda, Emma Cho, Jing Chen, Zhang Manpin et autres

Mucopolysaccharidosis IV A (MPS IVA) or Morquio A Syndrome is a lysosomal storage disorder, which results from a deficiency of N-acetylgalactosamine-6-sulfate sulfatase (GALNS) causing deposition of glycosaminoglycans, keratan sulfate and chondroitin-6-sulfate. Clinical manifestations include progressive skeletal dysplasia, and most patients become wheelchair-bound …

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Conditioning with rabbit versus horse ATG dramatically alters clinical outcomes in identical twins with severe aplastic anemia transplanted with the same allogeneic donor

Phuong Vo, Jeremy Pantin, Catalina Ramos, Lisa Cook et autres

Severe aplastic anemia (SAA) is a rare disorder leading to bone marrow failure, which if left untreated, is invariably fatal. Conventional therapies with immunosuppressive therapy or allogeneic hematopoietic stem cell transplantation (HSCT) are highly effective. HSCT can offer a greater outcome in …

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