Unusual setting of medical care, Rare disease, Congenital defects / diseases, Educational Purpose (only if useful for a systematic review or synthesis)
Alok Sharma, Hemangi Sane, Amruta Paranjape, Khushboo Bhagwanani, Nandini Gokulchandran, Prerna Badhe
Department of Medical Services and Clinical Research, NeuroGen Brain and Spine Institute, Mumbai, India
Am J Case Rep 2014; 15:128-134
Available online: 2014-03-28
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