Article
Longitudinal Analysis of Ocular Disease in Children with Mucopolysaccharidosis I after Hematopoietic Cell Transplantation.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation - 1 May 2020
van den Broek Brigitte T A, van Egmond-Ebbeling Michelle B, Achterberg Jens A, Boelens Jaap Jan, Vlessert Isa C, Prinsen Hubertus C M T, van Doorn Jaap, van Hasselt Peter M
Abstract excerpt
Corneal clouding, causing visual impairment, is seen in nearly all patients with mucopolysaccharidosis type 1 (MPS-1). Hematopoietic cell transplantation (HCT) is able to stabilize disease in many organs. Residual disease in several tissues is being increasingly recognized, however. Data on the effect of HCT on ocular disease in patients with MPS-1 are contradictory. With this study, we aim to clarify the...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.