Rationale : Spinal Muscular Atrophy (SMA) is the most common genetic disorder and presents the most common cause of infant mortality. To date, patient management is symptomatic and focuses on improvement of independence and treatment of complications. Stem cell therapy represents a novel therapeutic option for many neurological diseases. Presenting concerns of the patient: This patient with type III SMA presented with generalized hypotonia and muscle weakness with inability to raise hands and legs, support back or neck, in addition to respiratory distress. Diagnosis: Clinical examination showed hypotonia and loss of reflexes. Creatine kinase levelxxx, electrophysiologyxx. Interventions: Allogenic mesenchymal stem cells (MSCs) were injected in a dose of xxx intrathecally and a dose of xxx injected systemically. Outcomes: The patient showed improvement of GFM score and upgrading of the GFMC grade from Grade V to Grade III in 3 months. Improved quality of life was reflected in improvement of the PEDI scores. Improvement was noticed in respiration. No complications were encountered. Improvement was maintained until date. Conclusions: Allogenic MSC therapy may present a new therapeutic strategy for SMA patients. Controlled clinical trials are recommended to document the safety and efficacy of the procedure.
| Published in |
American Journal of Bioscience and Bioengineering (Volume 3, Issue 4-1)
This article belongs to the Special Issue Stem Cells for Neuro-Regeneration: Where Do We Stand |
| DOI | 10.11648/j.bio.s.2015030401.15 |
| Page(s) | 30-33 |
| Creative Commons |
This is an Open Access article, distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution and reproduction in any medium or format, provided the original work is properly cited. |
| Copyright |
Copyright © The Author(s), 2015. Published by Science Publishing Group |
Neuroregeneration, Spinal Muscle Atrophy, Mesenchymal Stem Cells
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APA Style
Abo Elkheir W., Gabr H., Salah Y. (2015). Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report. American Journal of Bioscience and Bioengineering, 3(4-1), 30-33. https://doi.org/10.11648/j.bio.s.2015030401.15
ACS Style
Abo Elkheir W.; Gabr H.; Salah Y. Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report. Am. J. BioSci. Bioeng. 2015, 3(4-1), 30-33. doi: 10.11648/j.bio.s.2015030401.15
AMA Style
Abo Elkheir W., Gabr H., Salah Y. Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report. Am J BioSci Bioeng. 2015;3(4-1):30-33. doi: 10.11648/j.bio.s.2015030401.15
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Download@article{10.11648/j.bio.s.2015030401.15,
author = {Abo Elkheir W. and Gabr H. and Salah Y.},
title = {Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report},
journal = {American Journal of Bioscience and Bioengineering},
volume = {3},
number = {4-1},
pages = {30-33},
doi = {10.11648/j.bio.s.2015030401.15},
url = {https://doi.org/10.11648/j.bio.s.2015030401.15},
eprint = {https://article.sciencepublishinggroup.com/pdf/10.11648.j.bio.s.2015030401.15},
abstract = {Rationale : Spinal Muscular Atrophy (SMA) is the most common genetic disorder and presents the most common cause of infant mortality. To date, patient management is symptomatic and focuses on improvement of independence and treatment of complications. Stem cell therapy represents a novel therapeutic option for many neurological diseases. Presenting concerns of the patient: This patient with type III SMA presented with generalized hypotonia and muscle weakness with inability to raise hands and legs, support back or neck, in addition to respiratory distress. Diagnosis: Clinical examination showed hypotonia and loss of reflexes. Creatine kinase levelxxx, electrophysiologyxx. Interventions: Allogenic mesenchymal stem cells (MSCs) were injected in a dose of xxx intrathecally and a dose of xxx injected systemically. Outcomes: The patient showed improvement of GFM score and upgrading of the GFMC grade from Grade V to Grade III in 3 months. Improved quality of life was reflected in improvement of the PEDI scores. Improvement was noticed in respiration. No complications were encountered. Improvement was maintained until date. Conclusions: Allogenic MSC therapy may present a new therapeutic strategy for SMA patients. Controlled clinical trials are recommended to document the safety and efficacy of the procedure.},
year = {2015}
}
TY - JOUR T1 - Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report AU - Abo Elkheir W. AU - Gabr H. AU - Salah Y. Y1 - 2015/06/30 PY - 2015 N1 - https://doi.org/10.11648/j.bio.s.2015030401.15 DO - 10.11648/j.bio.s.2015030401.15 T2 - American Journal of Bioscience and Bioengineering JF - American Journal of Bioscience and Bioengineering JO - American Journal of Bioscience and Bioengineering SP - 30 EP - 33 PB - Science Publishing Group SN - 2328-5893 UR - https://doi.org/10.11648/j.bio.s.2015030401.15 AB - Rationale : Spinal Muscular Atrophy (SMA) is the most common genetic disorder and presents the most common cause of infant mortality. To date, patient management is symptomatic and focuses on improvement of independence and treatment of complications. Stem cell therapy represents a novel therapeutic option for many neurological diseases. Presenting concerns of the patient: This patient with type III SMA presented with generalized hypotonia and muscle weakness with inability to raise hands and legs, support back or neck, in addition to respiratory distress. Diagnosis: Clinical examination showed hypotonia and loss of reflexes. Creatine kinase levelxxx, electrophysiologyxx. Interventions: Allogenic mesenchymal stem cells (MSCs) were injected in a dose of xxx intrathecally and a dose of xxx injected systemically. Outcomes: The patient showed improvement of GFM score and upgrading of the GFMC grade from Grade V to Grade III in 3 months. Improved quality of life was reflected in improvement of the PEDI scores. Improvement was noticed in respiration. No complications were encountered. Improvement was maintained until date. Conclusions: Allogenic MSC therapy may present a new therapeutic strategy for SMA patients. Controlled clinical trials are recommended to document the safety and efficacy of the procedure. VL - 3 IS - 4-1 ER -
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