Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: Clinical, biochemical, and pathological improvements

@inproceedings{Yasuda2015LongtermFO,
  title={Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: Clinical, biochemical, and pathological improvements},
  author={Eriko Yasuda and William G. Mackenzie and Kristen D. Ruhnke and Tsutomu Shimada and Robert W. Mason and Jozef Zustin and Paul Langlie Martin and Mihir M. Thacker and Tadao Orii and Yoshimichi Sai and Shunji Tomatsu},
  booktitle={Molecular genetics and metabolism reports},
  year={2015}
}
Mucopolysaccharidosis type I (MPS I; Hurler Syndrome) is a lysosomal storage disease caused by a deficiency of the enzyme α-L-iduronidase which affects multiple organs such as central nervous system (CNS), skeletal system, and physical appearance. Hematopoietic stem cell transplantation (HSCT) is recommended as a primary therapeutic option at an early stage… CONTINUE READING