Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report
American Journal of Bioscience and Bioengineering
Volume 3, Issue 4-1, July 2015, Pages: 30-33
Received: Jun. 9, 2015;
Accepted: Jun. 10, 2015;
Published: Jun. 30, 2015
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Abo Elkheir W., Immunology Department, Military Medical Academy, Cairo, Egypt
Gabr H., Clinical Pathology Department, Faculty of Medicine, Cairo University, Cairo Egypt
Salah Y., Anaesthesia Department, Military Medical Academy, Cairo, Egypt
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.
Abo Elkheir W.,
Allogenic Mesenchymal Stromal Cell Therapy for Type III Spinal Muscular Atrophy: Case Report, American Journal of Bioscience and Bioengineering. Special Issue:Stem Cells for Neuro-Regeneration: Where Do We Stand.
Vol. 3, No. 4-1,
2015, pp. 30-33.
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