Article
The effect of haemopoietic stem cell transplantation on the ocular phenotype in mucopolysaccharidosis type I (Hurler).
Acta ophthalmologica - 1 Aug 2018
Javed Ahmed, Aslam Tariq, Jones Simon A, Mercer Jean, Tyler Karen, Church Heather, Ghosh Arunabha, Wynn Robert, Sornalingam Krishanthy, Ashworth Jane
Abstract excerpt
PURPOSE: To determine whether the ocular phenotype in patients with mucopolysaccharidosis type I (MPSI) Hurler is affected by the efficacy of previous haemopoietic stem cell transplantation (HSCT). DESIGN: A retrospective cohort study of patients with MPSI who had undergone treatment with HSCT. METHODS: Ocular phenotype was documented for each patient and compared to levels of biomarkers representing efficacy of...
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