Early enzyme replacement therapy enables a successful hematopoietic stem cell transplantation in mucopolysaccharidosis type IH: Divergent clinical outcomes in two Japanese siblings
Sign Up to like & getrecommendations! Published in 2019 at "Brain and Development"
DOI: 10.1016/j.braindev.2019.01.008
Abstract: Mucopolysaccharidosis type IH (MPS IH, Hurler syndrome) is a progressive, multisystem autosomal recessive lysosomal storage disorder resulting in the consequent accumulation of glycosaminoglycans. It is well recognized that early hematopoietic stem cell transplantation (HSCT) prevents… read more here.
Keywords: mucopolysaccharidosis type; two japanese; cell transplantation; stem cell ... See more keywords
Divergent clinical presentations and outcomes among children and adolescents with Kaposi sarcoma in Malawi and Tanzania.
Sign Up to like & getrecommendations! Published in 2023 at "HIV medicine"
DOI: 10.1111/hiv.13455
Abstract: The Kaposi sarcoma (KS) T0 versus T1 staging classification does not address the unique clinical features of paediatric KS in human gammaherpesvirus 8 (HHV‐8) endemic regions of Africa. This study seeks to define patterns of… read more here.
Keywords: clinical presentations; divergent clinical; among children; outcomes among ... See more keywords