Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome

@article{CoxBrinkman2006HaematopoieticCT,
  title={Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome},
  author={Josanne Cox-Brinkman and J-J Boelens and J Edmund Wraith and Anne O'meara and Paul A. Veys and Frits A. Wijburg and N M Wulffraat and Robert Francis Wynn},
  journal={Bone Marrow Transplantation},
  year={2006},
  volume={38},
  pages={17-21}
}
Hurler syndrome (MPS 1H) is the severe form of mucopolysaccharidosis type 1 (MPS 1). Haematopoietic cell transplantation (HCT) is the treatment of choice, but carries a high incidence of graft failure and morbidity. The use of enzyme replacement therapy (ERT) might improve the clinical signs and symptoms before HCT, resulting in less transplantation-related complications. Moreover, clearance of glycosaminoglycans (GAG's) from the bone marrow might improve engraftment. Twenty-two patients with… CONTINUE READING
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