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28 March 2014 : Case report  India

Autologous bone marrow mononuclear cell transplantation in Duchenne muscular dystrophy

Unusual setting of medical care, Rare disease, Congenital defects / diseases, Educational Purpose (only if useful for a systematic review or synthesis)

Alok SharmaABCDEFG, Hemangi SaneABCDEFG, Amruta ParanjapeABCDEF, Khushboo BhagwananiBE, Nandini GokulchandranBG, Prerna BadheBG

DOI: 10.12659/AJCR.890078

Am J Case Rep 2014; 15:128-134

Abstract

BACKGROUND: Duchenne muscular dystrophy (DMD) is a fatal, genetic, progressive, degenerating muscle disorder. Current treat- ment options are palliative. Newer options of cellular therapy promise to alter the disease process. Preclinical studies have successfully tested myogenic, neurogenic potential and dystrophin expression of bone marrow mononuclear cells. 

CASE REPORT: We treated a 9-year-old boy suffering from DMD with serial autologous bone marrow mononuclear cell trans- plantations followed by multidisciplinary rehabilitation. Brooke-Vignos score was 10 and he was wheelchair- bound. Over 36 months, gradual progressive improvement was noticed in muscle strength, ambulation with assistive devices, fine motor movements, Brooke-Vignos score, and functional independence measure score. Nine months after the transplantation, electromyography findings showed development of new normal motor unit potentials of the vastus medialis muscle. 

CONCLUSIONS: Magnetic resonance imaging scan of musculoskeletal systems showed no increase in fatty infiltration. This case report provides early investigative findings or the restorative effects of cellular therapy in DMD. 

Keywords: Stem Cell Transplantation, Autologous bone marrow mono nuclear cells, Electromyography, Muscular Dystrophy, Duchenne - congenital

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American Journal of Case Reports eISSN: 1941-5923
American Journal of Case Reports eISSN: 1941-5923