دورية أكاديمية
Case study of two Iraqi patients with Mucopolysaccharidosis (Hurler syndrome 'type I' and Maroteaux-Lamy syndrome 'type VI') treated with Hematopoietic Stem Cell Transplantation (HSCT)
| العنوان: | Case study of two Iraqi patients with Mucopolysaccharidosis (Hurler syndrome 'type I' and Maroteaux-Lamy syndrome 'type VI') treated with Hematopoietic Stem Cell Transplantation (HSCT) |
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| المؤلفون: | Furqan M. Abdulelah, Mohammed M. Mohammed, Rabab Hassan Baaker |
| المصدر: | Al-Mustansiriyah Journal of Pharmaceutical Sciences, Vol 22, Iss 4 (2023) |
| بيانات النشر: | College of Pharmacy / Mustansiriyah University, 2023. |
| سنة النشر: | 2023 |
| المجموعة: | LCC:Pharmacy and materia medica |
| مصطلحات موضوعية: | Hematopoietic Stem Cells Transplantation, Matched donor, Hurler syndrome, Maroteaux-Lamy syndrome., Pharmacy and materia medica, RS1-441 |
| الوصف: | Mucopolysaccharidosis I (MPS I) or Hurler and Mucopolysaccharidosis VI (MPS VI) or Maroteaux-Lamy syndrome are infrequent genetic disorder inherited as an autosomal recessive disease attributed to genetic variants genetic variant causing α-L iduronidase (IDUA) and arylsulfatase B (ARSB)enzyme deficiency, respectively. Here, two cases of children suffering from MPS disorder were described, the first case was MPS I while the second case was MPS VI and both cases were treated with allogenic Hematopoietic Stem Cell Transplantation approach in order to limit skeletal deterioration and retard neurocognitive alterations and hence, improve the quality of life of affected children. Following Transplantations outcomes reveal a full engraftment of donor cells as well as improvement of recipient enzymatic activity, enzyme replacement therapy post-transplantation will augment transplantation clinical outcomes. Transplantation will be more successful if the disease diagnosed early before the severe irreversible symptoms ensue. |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 1815-0993 2959-183X |
| Relation: | https://ajps.uomustansiriyah.edu.iq/index.php/AJPS/article/view/958; https://doaj.org/toc/1815-0993; https://doaj.org/toc/2959-183X |
| DOI: | 10.32947/ajps.v22i4.958 |
| URL الوصول: | https://doaj.org/article/26d428542f4449a6b47f6ccf18c10f62 |
| رقم الأكسشن: | edsdoj.26d428542f4449a6b47f6ccf18c10f62 |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 18150993 2959183X |
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| DOI: | 10.32947/ajps.v22i4.958 |