The Effects of Enzyme Replacement Therapy in Patients with Mucopolysaccharidosis Type 1: A Case Series Study
Background: Mucopolysaccharidosis type I (MPS1) is caused by mutations in the gene which encodes the enzyme alpha-L-iduronidase. Deficiency of this enzyme causes a range of clinical symptoms in patients. The main treatment for MPS1 is hematopoietic stem cell transplantation. But its morbidity and mo...
| Main Authors: | , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Mashhad University of Medical Sciences
2022-04-01
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| Series: | International Journal of Pediatrics |
| Subjects: | |
| Online Access: | https://ijp.mums.ac.ir/article_19739_4b5f9f475025b99974564e3a2c70be75.pdf |